American Journal of Medical Genetics Part A - 2021

571 articles | Last updated: 2025-12-03 14:12:55
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T A T A T A T A
Identification of the first <scp>promoter‐specific gain‐of‐function <i>SOX9</i></scp> missense variant (p.<scp>E50K</scp>) in a patient with 46,<scp>XX</scp> ovotesticular disorder of sex development
Chondrodysplasia and growth failure in children after early hematopoietic stem cell transplantation for non‐oncologic disorders
Disorder of sex development associated with a novel homozygous nonsense mutation in <scp><i>COG6</i></scp> expands the phenotypic spectrum of <scp>COG6‐CDG</scp>
Insufficient development of vessels and alveoli in lungs of infants with trisomy 18—Features of pulmonary histopathological findings from lung biopsy
Epilepsy and electroencephalogram evolution in <scp><i>YWHAG</i></scp> gene mutation: A new phenotype and review of the literature
Ayme gripp syndrome in an Indian patient
Congenital heart defects in molecularly confirmed <scp>KBG</scp> syndrome patients
A de novo <scp><i>ACTB</i></scp> gene pathogenic variant in identical twins with phenotypic variation for hydrops and jejunal atresia
The longest reported sibling survivors of a severe form of congenital myasthenic syndrome with the <scp><i>ALG14</i></scp> pathogenic variant
Efficacy of virtual and asynchronous teaching of c<scp>omputer‐assisted</scp> diagnosis of genetic diseases seen in clinics
Corrigendum Presacral Neuroendocrine Tumors Associated with the Currarino Syndrome. Am J Med Genet A. 2021;185(5):1582–1588. Doi:10.1002/ajmg.a.62145
Craniosynostosis is a feature of Costello syndrome
Corrigendum The phenotypic spectrum of AMER1‐related osteopathia striata with cranial sclerosis: The first Canadian cohort. Am J Med Genet A. 2021;185(12):3793–3803. Doi:10.1002/ajmg.a.62452
Growth in Joubert syndrome: Growth curves and physical measurements with correlation to genotype and hepatorenal disease in 170 individuals
Corrigendum Parental mosaicism in de novo neurodevelopmental diseases. Am J Med Genet A. 2021;<scp>185A</scp>(7):2119–2125. Doi:10.1002/ajmg.a.62174
Corrigendum Candidate Gene Locus for PHACE Syndrome. Am J Med Genet A. 2012;158(6):1363–1367. Doi:10.1002/ajmg.a.35341
<scp>Kohlschütter–Tönz</scp> syndrome: Case report with novel feature and detailed review of features associated with <scp><i>ROGDI</i></scp> variants
Extending the phenotype of posterior column ataxia with retinitis pigmentosa caused by variants in <scp><i>FLVCR1</i></scp>
Truncating and zinc‐finger variants in <scp><i>GLI2</i></scp> are associated with hypopituitarism
A homozygous missense variant in the <i>MLC1</i> gene underlies megalencephalic leukoencephalopathy with subcortical cysts in large kindred: Heterozygous carriers show seizure and mild motor function
Publication schedule for 2022
Cover Image, Volume 188A, Number 1, January 2022
Use of the Vineland‐3, a measure of adaptive functioning, in <scp>CLN3</scp>
Table of Contents, Volume 188A, Number 1, January 2022
ACMG Updates Guidance on Carrier Screening
Identifying Digenic Disease Genes with Machine Learning
In This Issue
A novel missense variant of <scp><i>SCN4A</i></scp> co‐segregates with congenital essential tremor in a consanguineous Kurdish family Kurdish
Bone tissue homeostasis and risk of fractures in Costello syndrome: A 4‐year follow‐up study
<scp>Zimmermann–Laband</scp> syndrome in monozygotic twins with a mild neurobehavioral phenotype lacking gingival overgrowth—A case report of a novel <scp><i>KCNN3</i></scp> gene variant
Prenatal diagnosis of a likely pathogenic variant in <i>ZBTB18</i>: Natural evolution of fetal phenotype including the long bones and corpus callosum
An examination of adaptive behavior and functional outcomes in adults with 22q11.2 deletion syndrome: A parental perspective
Identification and validation of a novel pathogenic variant in <scp><i>GDF2</i></scp> (<scp>BMP9</scp>) responsible for hereditary hemorrhagic telangiectasia and pulmonary arteriovenous malformations
Rare presentation of <scp><i>FDX2</i>‐related</scp> disorder and untargeted global metabolomics findings
Further delineation of phenotypic spectrum of <scp><i>SCN2A</i></scp>‐related disorder
Vein of Galen aneurysm, dilated cardiomyopathy, and slender habitus in a patient with a recurrent pathogenic variant in <scp><i>PACS2</i></scp>
Tibia hemimelia in a patient with <scp>CHARGE</scp> syndrome: A rare but recurrent phenomenon
Clinical and molecular response to dasatinib in an adult patient with Penttinen syndrome
A novel variant in the dystonin gene causing hereditary sensory autonomic neuropathy type <scp>VI</scp> in a male infant: Case report and literature review
8p23.1 <scp>deletion</scp>: <scp>Look out for left ventricular hypertrabeculation and not only congenital heart diseases</scp>. <scp>Single</scp>‐<scp>center experience and literature revision</scp>
Giant axonal neuropathy (<scp>GAN</scp>) in an 8‐year‐old girl caused by a homozygous pathogenic splicing variant in <scp><i>GAN</i></scp> gene
High‐level mosaic monosomy 21 in a 13‐year‐old girl: Case report and review of the literature
Sleep problems in fragile X syndrome: Cross‐sectional analysis of a large clinic‐based cohort
Improving survival in patients with trisomy 18
Further supporting <scp><i>SMARCC2</i></scp>‐related neurodevelopmental disorder through exome analysis and reanalysis in two patients
Biallelic <i>TERT</i> variant leads to Hoyeraal–Hreidarsson syndrome with additional dyskeratosis congenita findings
The association between mosaicism type and cognitive and behavioral functioning among males with fragile X syndrome
Cornelia de Lange syndrome and the Cohesin complex: Abstracts from the 9th Biennial Scientific and Educational Virtual Symposium 2020
A mutation in the neonatal isoform of <i>SCN2A</i> causes neonatal‐onset epilepsy
<i>Ectodysplasin</i> pathogenic variants affecting the furin‐cleavage site and unusual clinical features define X‐linked hypohidrotic ectodermal dysplasia in India
Psychomotor development in infants and young children with Down syndrome—A prospective, repeated measure, post‐hoc analysis
A homozygous <i>CAP2</i> pathogenic variant in a neonate presenting with rapidly progressive cardiomyopathy and nemaline rods
Intracranial venous malformation masquerading as a meningioma in <i>PI3KCA</i>‐related overgrowth spectrum disorder
An <scp><i>ACVR1</i><sup><i>R375P</i></sup></scp> pathogenic variant in two families with mild fibrodysplasia ossificans progressiva
<i>N</i>‐Acetylcysteine provides limited efficacy as treatment option for skin picking in <scp>Prader–Willi</scp> syndrome
Characterization of bone homeostasis in individuals affected by cardio‐facio‐cutaneous syndrome
22q11.2 duplications: Expanding the clinical presentation
Expanded phenotype of primary ciliary dyskinesia related to <scp><i>DRC1</i></scp> pathogenic variant with dysmorphisms and vascular anomalies
Report of two children with global developmental delay in association with de novo <scp><i>TLK2</i></scp> variant and literature review
Improved attention linked to sustained phenylalanine reduction in adults with early‐treated phenylketonuria
Leber congenital amaurosis as an initial manifestation in a Chinese patient with thiamine‐responsive megaloblastic anemia syndrome
An infant with congenital respiratory insufficiency and diaphragmatic paralysis: A novel <scp><i>BICD2</i></scp> phenotype?
Quantitative dissection of multilocus pathogenic variation in an Egyptian infant with severe neurodevelopmental disorder resulting from multiple molecular diagnoses
<i>First</i> case report of Penttinen syndrome from India
Sanfilippo syndrome type B: Analysis of patients diagnosed by the <scp>MPS</scp> Brazil Network
Case report of atypical Leigh syndrome in an adolescent male with novel biallelic variants in <scp><i>NDUFAF5</i></scp> and review of the natural history of <scp><i>NDUFAF5</i></scp>‐related disorders African American
De novo <scp><i>PBX1</i></scp> variant in a patient with glaucoma, kidney anomalies, and developmental delay: An expansion of the <scp>CAKUTHED</scp> phenotype
Extending the phenotype of <scp>DeSanto‐Shinawi</scp> syndrome: A case report and literature review
Dominant and recessive <scp><i>SLC12A2</i></scp>‐syndrome
Neurofibromatosis 1 in the setting of dual diagnosis: Diagnostic and management conundrums
Publication schedule for 2021
Cover Image, Volume 185A, Number 12, December 2021
Long‐Read Sequencing Could Increase Diagnosis Rates
The Road to Greater Diversity in the Genomics Workforce
Table of Contents, Volume 185A, Number 12, December 2021
In This Issue
Distinguishing severe phenotypes associated with pathogenic variants in <i>POLR3A</i>
Ocular findings in 22q11.2 deletion syndrome: A systematic literature review and results of a Dutch multicenter study
Homozygous microdeletion in the 11p13 region in the patient with isolated form of aniridia: New challenges in the genetic diagnostics of aniridia Polish
The transition to independence and adult care for women with Turner syndrome: Current status and priorities of 1338 women and parents
<scp><i>PHIP</i></scp> gene variants with protein modeling, interactions, and clinical phenotypes
A severe case of Bosch–<scp>Boonstra–Schaaf</scp> optic atrophy syndrome with a novel description of coloboma and septo‐optic dysplasia, owing to a start codon variant in the <scp><i>NR2F1</i></scp> g
Expanding the phenotypic and allelic spectrum of <scp><i>SMG8</i></scp>: Clinical observations reveal overlap with <i><scp>SMG9</scp>‐</i>associated disease trait
Corrigendum Re: Expanding the genotypic and phenotypic spectrum in a diverse cohort of 104 individuals with Wiedemann‐Steiner syndrome. Am J Med Genet A. 2021;185A(6):1649–1665. Doi:10.1002/ajmg.a.621
Psychological predictors of advanced cancer patients’ preferences for return of results from comprehensive tumor genomic profiling
A novel <scp><i>DLL4</i></scp> mutation in <scp>Adams–Oliver</scp> syndrome with absence of the right pulmonary artery in newborn Thai
Identification and functional study of <scp><i>FOXC1</i></scp> variants in Chinese families with glaucoma
A new case of <scp>Turnpenny‐Fry</scp> syndrome
Pseudoachondroplasia: Phenotype and genotype in 11 Indian patients Indian ethnicity
Clinical features of a male with a <scp><i>USP9X</i></scp> variant associated with intellectual disability: A case study and review of reported cases
Adolescents and young adults with neurofibromatosis type 1: A descriptive study of adaptive functioning
Pulmonary vascular resistance and compliance in individuals with trisomy 18
N‐methyl‐d‐aspartate (NMDA) receptor genetics: The power of paralog homology and protein dynamics in defining dominant genetic variants
Direct hyperbilirubinemia and cholestasis in trisomy 13 and 18
A disease‐causing variant in <scp><i>HNRNPH2</i></scp> inherited from an unaffected mother with skewed X‐inactivation
<scp><i>PUS3</i></scp>‐related disorder: Report of a novel patient and delineation of the phenotypic spectrum
<scp>Cardiovascular findings in Williams–Beuren Syndrome</scp>: Experience of a single center with 127 cases
Expanding the genetic and phenotypic spectrum of <scp><i>CHD2</i></scp>‐related disease: From early neurodevelopmental disorders to adult‐onset epilepsy
A recurrent rare intronic variant in <scp><i>CAPN3</i></scp> alters <scp>mRNA</scp> splicing and causes autosomal recessive limb‐girdle muscular dystrophy‐1 in three Pakistani pedigrees Pakistani origin
Descriptive and risk factor analysis of infantile cataracts: National Birth Defects Prevention Study, 2000–2011
Variable clinical severity in <scp>TANGO2</scp> deficiency: Case series and literature review
The <scp><i>RRAS2</i></scp> pathogenic variant p.<scp>Q72L</scp> produces severe Noonan syndrome with hydrocephalus: A case report
Genotype‐cardiac phenotype correlations in a large single‐center cohort of patients affected by RASopathies: Clinical implications and literature review
Aortic root dilatation and dilated cardiomyopathy in an adult with <scp>Tatton‐Brown‐Rahman</scp> syndrome
The novel and recurrent variants in exon 31 of <scp><i>CREBBP</i></scp> in Japanese patients with <scp>Menke–Hennekam</scp> syndrome Japanese
An <i>LMNA</i> synonymous variant associated with severe dilated cardiomyopathy: Case report
Publication schedule for 2021
In This Issue
Heritable disorders of oxygen sensing
A novel <scp><i>MBTPS2</i></scp> variant associated with <scp>BRESHECK</scp> syndrome impairs <scp>sterol‐regulated</scp> transcription and the endoplasmic reticulum stress response
Cover Image, Volume 185A, Number 11, November 2021
Koolen‐de Vries syndrome in a 63‐year‐old woman: Report of the oldest patient and a review of the adult phenotype
A Promising Option for ADA‐SCID Patients
ACMG Releases Guidelines for Exome and Genome Sequencing for Pediatric Patients
Table of Contents, Volume 185A, Number 11, November 2021
Rare neurological manifestations in a Saudi Arabian patient with <scp>Ehlers–Danlos</scp> syndrome and a novel homozygous variant in the <scp><i>TNXB</i></scp> gene
Functioning and well‐being in older children and adolescents with achondroplasia: A qualitative study
Can artificial intelligence save medical genetics?
Genomic basis of syndromic short stature in an Algerian patient cohort
Isolated cytokine‐enriched pericardial effusion: A likely key feature for <scp>Aymé‐Gripp</scp> syndrome
Causes of death in patients with Down syndrome in 2014–2016: A population study in Japan
D‐bifunctional protein deficiency caused by splicing variants in a neonate with severe peroxisomal dysfunction and persistent hypoglycemia
<scp><i>INTU</i></scp>‐related oral‐facial‐digital syndrome <scp>XVII</scp>: Clinical spectrum of a rare disorder
A patient with compound heterozygosity of <scp><i>SMPD4</i></scp>: Another example of utility of exome‐based copy number analysis in autosomal recessive disorders Japanese
Novel <scp><i>DIP2C</i></scp> gene splicing variant in an individual with focal infantile epilepsy
Vanishing basal ganglia in <scp><i>ATP1A3</i></scp>‐related polymicrogyria
A homozygous <scp><i>GRIN1</i></scp> null variant causes a more severe phenotype of early infantile epileptic encephalopathy
Clinical manifestations of patients with <scp><i>GDF2</i></scp> mutations associated with hereditary hemorrhagic telangiectasia type 5
A novel homozygous synonymous variant further expands the phenotypic spectrum of <i><scp>POLR3A</scp>‐</i>related pathologies
Expanding on the phenotypic spectrum of<scp>Woodhouse‐Sakati</scp>syndrome due to founder pathogenic variant in<scp><i>DCAF17</i></scp>: Report of 58 additional patients from Qatar and literature revi
Extending the clinical phenotype of <scp><i>SPTAN1</i></scp>: From <scp>DEE5</scp> to migraine, epilepsy, and subependymal heterotopias without intellectual disability
Bi‐allelic <scp><i>PAGR1</i></scp> variants are associated with microcephaly and a severe neurodevelopmental disorder: Genetic evidence from two families Ashkenazi Jewish families
Three decades of the Human Genome Organization
A monoallelic<scp><i>SEC23A</i></scp>variant<scp>E599K</scp>associated with<scp>cranio‐lenticulo‐sutural</scp>dysplasia
<scp><i>CTNNB1</i></scp>‐related neurodevelopmental disorder in a Chinese population: A case series
Correspondence on “Disorder of sex development associated with a novel homozygous nonsense mutation in <scp><i>COG6</i></scp> expands the phenotypic spectrum of <scp><i>COG6</i>‐CDG</scp>”
Parental perceptions of genetic testing for children with autism spectrum disorders
<scp><i>NDE1</i></scp>‐related disorders: A recurrent <scp><i>NDE1</i></scp> pathogenic variant causing Lissencephaly 4 can also be associated with microhydranencephaly
Mesenteric cysts, lymphatic leak, and cerebral cavernous malformation in a proband with <scp><i>KRIT1</i></scp>‐related disease
Expanding the phenotypic spectrum of cardiospondylocarpofacial syndrome: From a detailed clinical and radiological observation of a boy with a novel missense variant in <scp><i>MAP3K7</i></scp> Asian
Review of 37 patients with <scp><i>SOX2</i></scp> pathogenic variants collected by the Anophthalmia/Microphthalmia Clinical Registry and <scp>DNA</scp> research study
A nonsense variant in the second exon of the canonical transcript of <scp><i>NSD1</i></scp> does not cause Sotos syndrome
Genomic analysis of “microphenotypes” in epilepsy
A homozygous <scp><i>ROR2</i></scp> variant in a family with atypical Robinow syndrome and tetramelic transverse deficiency of autopods
Robin sequence without cleft palate: Genetic diagnoses and management implications
<scp><i>GGCX</i></scp>‐related congenital combined vitamin K‐dependent clotting factors deficiency‐1: Description of a fetus with chondrodysplasia punctata
Robinow syndrome in an extremely preterm infant: Novel homozygous <scp><i>ROR2</i></scp> variant detected by rapid exome sequencing
Novel hemizygous loss‐of‐function variant in <scp><i>NONO</i></scp> identified in a South African boy
Impact of the coronavirus pandemic on mental health and health care in adults with neurofibromatosis: Patient perspectives from an online survey
Nicotinamide nucleotide transhydrogenase mutation analysis in Chinese patients with thyroid dysgenesis
Paternal retraction of a fragile X allele to normal size, showing normal function over two generations
Digital vascular lesions detected by transillumination
Heterozygous Missense Variant in <scp><i>EIF6</i></scp> gene: a novel form of <scp>Shwachman‐Diamond</scp> Syndrome?
Mendelian disease research in the Plain populations of Lancaster County, Pennsylvania
Manifestation of epilepsy in a patient with <scp><i>EED</i></scp>‐related overgrowth (<scp>Cohen–Gibson</scp> syndrome)
A phenotypic expansion of <scp><i>TRNT1</i></scp> associated sideroblastic anemia with immunodeficiency, fevers, and developmental delay
A <i>de novo</i><scp><i>CSDE1</i></scp> variant causing neurodevelopmental delay, intellectual disability, neurologic and psychiatric symptoms in a child of consanguineous parents Turkish origin
A truncating <scp><i>NRIP1</i></scp> variant in an Arabic family with congenital anomalies of the kidneys and urinary tract Arabic family
Optogenetics Shows Promise in Landmark Study
Cover Image, Volume 185A, Number 10, October 2021
Publication schedule for 2021
Genetic and phenotypic heterogeneity in <scp>KIAA0753</scp>‐related ciliopathies
Table of Contents, Volume 185A, Number 10, October 2021
In This Issue
Tabula Sapiens: An Atlas of Single‐Cell Gene Expression
The rate of secondary genomic findings in the Saudi population
Heterozygous variants in <scp><i>ZBTB7A</i></scp> cause a neurodevelopmental disorder associated with symptomatic overgrowth of pharyngeal lymphoid tissue, macrocephaly, and elevated fetal hemoglobin
Victor <scp>McKusick</scp> and his role in the founding of the European School of Genetic Medicine
Gain in growth after surgical repair of congenital heart disease among children with Down syndrome
Cross‐sectional and longitudinal findings in patients with proximal 22q11.2 duplication: A retrospective chart study
Identification of a novel <scp><i>TBX5</i></scp> c.755 + 1 G &gt; A variant and related pathogenesis in a family with <scp>Holt–Oram</scp> syndrome
Using Online Mendelian Inheritance in Man in low‐ and middle‐income countries
Medical, welfare, and educational challenges and psychological distress in parents caring for an individual with 22q11.2 deletion syndrome: A cross‐sectional survey in Japan
The Rizzoli Multiple Osteochondromas Classification revised: describing the phenotype to improve clinical practice
Expanding the phenotype: Four new cases and hope for treatment in <scp>Bachmann‐Bupp</scp> syndrome
<scp><i>PIEZO1</i></scp>‐gene gain‐of‐function mutations with lower limb lymphedema onset in an adult: Clinical, scintigraphic, and noncontrast magnetic resonance lymphography findings
Current state of the art in treatment of Mendelian disease: Skeletal dysplasias
Congenital heart defects and copy number variants associated with neurodevelopmental impairment
Esophageal atresia/tracheoesophageal fistula and proximal symphalangism in a patient with a <scp><i>NOG</i></scp> nonsense mutation
The youngest pair of siblings with Mucopolysaccharidosis type <scp>IVA</scp> to receive enzyme replacement therapy to date: A case report
The first <scp>post‐natal</scp> clinical description of true mosaic complete tetrasomy 21: A case report
Diverse clinical manifestations of Cantú syndrome: The first case series in Vietnam
A novel pathogenic variant p.<scp>Asp797Val</scp> in <scp><i>IFIH1</i></scp> in a Japanese boy with overlapping <scp>Singleton‐Merten</scp> syndrome and <scp>Aicardi‐Goutières</scp> syndrome
The burden of pathogenic variants in clinically actionable genes in a founder population Amish; Old Order Amish; founder population
A novel microdeletion upstream of <scp><i>HOXD13</i></scp> in a Chinese family with synpolydactyly
A novel homozygous mutation in the human <scp><i>ALG12</i></scp> gene results in an aberrant profile of oligomannose N‐glycans in patient's serum Slovak
Antenatal diagnostic dilemma in a pseudodominant pedigree with lamin‐<scp>B</scp> receptor (<scp>LBR</scp>)‐related regressive spondylometaphyseal dysplasia
Hartnup disease presenting as hereditary spastic paraplegia and severe peripheral neuropathy
Victor Almon <scp>McKusick</scp>: In the footsteps of Mendel and Osler
A recurrent de novo variant supports <scp><i>KCNC2</i></scp> involvement in the pathogenesis of developmental and epileptic encephalopathy
Expanding the phenotype of <scp><i>ASXL3</i></scp>‐related syndrome: A comprehensive description of 45 unpublished individuals with inherited and de novo pathogenic variants in <scp><i>ASXL3</i></scp>
Ancestral patterns of recessive dystrophic epidermolysis bullosa mutations in Hispanic populations suggest sephardic ancestry shared European and American origins of RDEB mutations; European origins; Hispanic populations; Sephardic ancestry; American origins
Deep intronic variant in the <scp><i>ARSB</i></scp> gene as the genetic cause for Maroteaux–Lamy syndrome (<scp>MPS VI</scp>) Bedouin origin
Toward precision medicine in vascular connective tissue disorders
Magnitude of Mendelian versus complex inheritance of rare disorders
Toward better characterization of restricted and repetitive behaviors in individuals with germline heterozygous <scp><i>PTEN</i></scp> mutations
The phenotypic spectrum of <scp><i>AMER1</i></scp>‐related osteopathia striata with cranial sclerosis: The first Canadian cohort
Survival outcomes of very low birth weight infants with trisomy 18
Novel cases of pediatric sudden cardiac death secondary to <scp><i>TRDN</i></scp> mutations presenting as long <scp>QT</scp> syndrome at rest and catecholaminergic polymorphic ventricular tachycardia Iranian population
The experiences and support needs of siblings of people with mucopolysaccharidosis
Twenty‐year follow‐up of the facial phenotype of Brazilian patients with Sotos syndrome
Whole genome sequencing identifies pathogenic <scp><i>RNU4ATAC</i></scp> variants in a child with recurrent encephalitis, microcephaly, and normal stature
Clan genomics: From <scp>OMIM</scp> phenotypic traits to genes and biology
Victor McKusick and his short course
Biallelic <i>ITGB4</i> variants in familial pyloric atresia without epidermolysis bullosa: Report of two families with five siblings
Cover Image, Volume 185A, Number 9, September 2021
Table of Contents, Volume 185A, Number 9, September 2021
Publication schedule for 2021
Age at and indication for diagnosis of Turner syndrome in the pediatric population
Neuroimaging in Kabuki syndrome and another <scp><i>KMT2D</i></scp>‐related disorder
Kleefstra syndrome: Recurrence in siblings due to a paternal mosaic mutation
Exome sequencing of child–parent trios with bladder exstrophy: Findings in 26 children
Expanding the phenotypic spectrum of Mendelian connective tissue disorders to include prominent kidney phenotypes
Expanding the <scp><i>KIF4A</i></scp>‐associated phenotype
Auditory and olfactory findings in patients with <scp><i>USH2A</i></scp>‐related retinal degeneration—Findings at baseline from the rate of progression in <scp><i>USH2A</i></scp>‐related retinal degen
Dysautonomia in hypermobile <scp>Ehlers–Danlos</scp> syndrome and hypermobility spectrum disorders is associated with exercise intolerance and cardiac atrophy
<scp>ZTTK</scp> syndrome: Clinical and molecular findings of 15 cases and a review of the literature
Exome survey of individuals affected by <scp>VATER</scp>/<scp>VACTERL</scp> with renal phenotypes identifies phenocopies and novel candidate genes
Festschrift for Victor A. <scp>McKusick</scp> on the Centenary of his Birth: Introduction
<scp><i>PLXNA2</i></scp> as a candidate gene in patients with intellectual disability
Psychiatric disorders in individuals with neurofibromatosis 1 in Denmark: A nationwide register‐based cohort study
A novel likely pathogenic heterozygous <scp><i>HECW2</i></scp> missense variant in a family with variable expressivity of neurodevelopmental delay, hypotonia, and epileptiform <scp>EEG</scp> patterns
Low‐level mosaicism in tuberous sclerosis complex in four unrelated patients: Comparison of clinical characteristics and diagnostic pathways
Autophagic defects observed in fibroblasts from a patient with β‐propeller protein‐associated neurodegeneration
5q11.2 deletion syndrome revisited—Further narrowing of the smallest region of overlap for the main clinical characteristics of the syndrome
Four hypotrichosis families with mutations in the gene <scp><i>LSS</i></scp> presenting with and without neurodevelopmental phenotypes
Memories of Victor A. <scp>McKusick</scp>
The recurrent p.(<scp>Pro540Ser</scp>) <scp><i>MEN1</i></scp> genetic variant should be considered nonpathogenic: A case report
Homozygous <scp><i>GLI3</i></scp> variants observed in three unrelated patients presenting with syndromic polydactyly
Decline in gross motor skills in adult Rett syndrome; results from a Danish longitudinal study
Cleft palate morphology, genetic etiology, and risk of mortality in infants with Robin sequence
Facial recognition accuracy in photographs of Thai neonates with Down syndrome among physicians and the <scp>Face2Gene</scp> application
Exon skip‐inducing variants in <scp><i>FLNA</i></scp> in an attenuated form of frontometaphyseal dysplasia
Detailed clinical and radiological features of the first patient with <scp>Elsahy–Waters</scp> syndrome in East Asia
Targeted therapy with galantamine in a pediatric patient with 15q13.3 deletion syndrome
Terminal osseous dysplasia with pigmentary defects and cardiomyopathy caused by a novel <scp><i>FLNA</i></scp> variant
Bronchial angiofibroma in tuberous sclerosis complex: A case report and literature review
Somatic <scp><i>KRAS</i></scp> mutation affecting codon 146 in linear sebaceous nevus syndrome
Corrigendum to “<scp><i>HECW2</i></scp>‐related disorder in four Japanese patients. Am J Med Genet Part A. First published: 28 May 2021 https://doi.org/10.1002/ajmg.a.62363”
A celebration in honor of John M. Graham, Jr, <scp>MD</scp>, <scp>ScD</scp>
Juvenile xanthogranuloma: A possible diagnostic criterion for Neurofibromatosis type 1 in young children
Promising Results with Ex Vivo Lentiviral HSPC Gene Therapy in ADA‐SCID
Cover Image, Volume 185A, Number 8, August 2021
Table of Contents, Volume 185A, Number 8, August 2021
Publication schedule for 2021
Geneticist Workforce Faces Critical Shortage
Early developmental impact of sex chromosome trisomies on attention <scp>deficit‐hyperactivity</scp> disorder symptomology in young children
In This Issue
Second instance of co‐occurring 22q11.2 deletion syndrome and Williams syndrome
<scp><i>EPHB4</i></scp> mutation causes adult and adolescent‐onset primary lymphedema
Further heterogeneity in <scp>Silver–Russell</scp> syndrome: <scp><i>PLAG1</i></scp> deletion in association with a complex chromosomal rearrangement
Samia Temtamy
Delivering a new diagnosis of Down syndrome: Parent experience
Functional analysis of novel genetic variants of <i><scp>NKX2</scp>‐5</i> associated with nonsyndromic congenital heart disease
Central 22q11.2 deletion (<scp>LCR22 B‐D</scp>) in a fetus with severe fetal growth restriction and a mother with severe systemic lupus erythematosus: Further evidence of <scp><i>CRKL</i></scp> haploi
The genes of <scp>OMIM</scp>: A legacy of Victor <scp>McKusick</scp>
<scp>Singleton‐Merten</scp> syndrome: A rare cause of femoral head necrosis
Severe diarrhea in a 10‐year‐old girl with <scp>Aicardi–Goutières</scp> syndrome due to <scp><i>IFIH1</i></scp> gene mutation
Personal utility of genomic sequencing for infants with congenital deafness
Comprehensive investigation of the phenotype of <scp><i>MEF2C</i>‐related</scp> disorders in human patients: A systematic review
Expanding the clinical phenotype of <scp>RASopathies</scp> in 38 Turkish patients, including the rare <scp><i>LZTR1</i></scp>, <scp><i>RAF1</i></scp>, <scp><i>RIT1</i></scp> variants, and large deleti
The introduction of clinical genetic testing in Ethiopia: Experiences and lessons learned
Online Mendelian Inheritance in Man (<scp>OMIM</scp>®): Victor <scp>McKusick</scp>'s magnum opus
In Memoriam: “Holstein cows in Holstein.” Victor A. <scp>McKusick</scp>: 40 years of remembrance from Europe
Health care transition for individuals with Down syndrome: A needs assessment
Milestones in treatments for inborn errors of metabolism: Reflections on <scp><i>Where chemistry and medicine meet</i></scp>
Genome sequencing for detection of pathogenic deep intronic variation: A clinical case report illustrating opportunities and challenges
Broadening the phenotypic spectrum of <scp>Beta3GalT6</scp>‐associated phenotypes
Hoarse voice in children as the presenting feature of <scp><i>ECM1</i></scp>‐related lipoid proteinosis
Expansion of the clinical phenotype of <scp>GALE</scp> deficiency
Nonlethal presentations of <scp>CYP26B1</scp>‐related skeletal anomalies and multiple synostoses syndrome
Viewing Victor<scp>McKusick'</scp>s legacy through the lens of his bibliography
History of the methodology of disease gene identification
Anticipating the ethical, legal, and social implications of human genome research: An ongoing experiment
L‐carnitine supplementation for muscle weakness and fatigue in children with neurofibromatosis type 1: A Phase 2a clinical trial
Physical therapy treatment of hypermobile Ehlers–Danlos syndrome: A systematic review
Homozygous <scp><i>WNT9B</i></scp> variants in two families with bilateral renal agenesis/hypoplasia/dysplasia
A novel truncating variant in the <scp><i>FGD1</i></scp> gene associated with Aarskog–Scott syndrome in a family previously diagnosed with Tel Hashomer camptodactyly
Corrigendum Re: “Persistence of müllerian duct structures in a genetic male with distal monosomy 10q. Am J Med Genet A. 2015 Apr;<scp>167A</scp>(4):791–6. Doi:10.1002/ajmg.a.37014”
Hepatocellular carcinoma as a complication of Niemann‐Pick disease type <scp>C1</scp>
Wolfram Syndrome: Cracking the Code to Better Therapies
Genomic Analysis Reveals Cancer–Like Mutagenesis in Placental Tissues
In This Issue
Expanding the genetic landscape of oral‐facial‐digital syndrome with two novel genes Dominican ancestry
Table of Contents, Volume 185A, Number 7, July 2021
Publication schedule for 2021
Cover Image, Volume 185A, Number 7, July 2021
Application of the <scp>ACMG</scp>/<scp>NSGC</scp> genetic referral guidelines for hereditary renal cell carcinoma at the University of Miami, from 2014 to 2017
<scp><i>PHKA2</i></scp> variants expand the phenotype of phosphorylase B kinase deficiency to include patients with ketotic hypoglycemia only
Never quit on hills: John M. Graham, Jr. <scp>MD</scp>, <scp>ScD,</scp> as mentor
Mosaic <scp>RASopathy</scp> due to <scp><i>KRAS</i></scp> variant <scp>G12D</scp> with segmental overgrowth and associated peripheral vascular malformations
Adult diagnosis of congenital serine biosynthesis defect: A treatable cause of progressive neuropathy
Expanding the clinical and radiological phenotypes of leukoencephalopathy due to biallelic <scp><i>HMBS</i></scp> mutations
Spondyloepimetaphyseal dysplasia <scp>EXTL3‐deficient</scp> type: Long‐term <scp>follow‐up</scp> and review of the literature
Risk of sudden cardiac death in <scp><i>EXOSC5</i></scp>‐related disease
Possible underreporting of pathogenic variants in <scp><i>RAI1</i></scp> causing <scp>Smith–Magenis</scp> syndrome
Caregiver‐reported characteristics of children diagnosed with pathogenic variants in <scp><i>KDM5C</i></scp>
Medical genetics training in the <scp>COVID</scp>‐19 era: A resident's perspective
Proximal variants in <scp><i>CCND2</i></scp> associated with microcephaly, short stature, and developmental delay: A case series and review of inverse brain growth phenotypes
Atypical, severe hypertrophic cardiomyopathy in a newborn presenting Noonan syndrome harboring a recurrent heterozygous <scp><i>MRAS</i></scp> variant
Whole genome sequencing identifies a cryptic <scp><i>SOX9</i></scp> regulatory element duplication underlying a case of 46,<scp>XX</scp> ovotesticular difference of sexual development
A rare description of pure partial trisomy of 16q12.2q24.3 and review of the literature
Recurrent <scp><i>KCNT2</i></scp> missense variants affecting p.Arg190 result in a recognizable phenotype
Temporal trends and yield of clinical diagnostic genetic testing in adult neurology
The evolution of genetic counseling at Johns Hopkins Hospital and beyond
Demographic, clinical, and ancestry characterization of a large cluster of mucopolysaccharidosis IV A in the Brazilian Northeast region predominantly European parental ancestry; European paternal haplogroups R1a, R1b; 'Paternal ancestry
Confirmation of Ogden syndrome as an X‐linked recessive fatal disorder due to a recurrent <scp>NAA10</scp> variant and review of the literature
Behavioral and cognitive functioning in individuals with Cantú syndrome
Novel findings and expansion of phenotype in a mosaic <scp>RASopathy</scp> caused by somatic <scp><i>KRAS</i></scp> variants
Pneumonia and respiratory infection in Down syndrome: A 10‐year cohort analysis of inpatient and outpatient encounters across the lifespan
Human cytogenetics at Johns Hopkins Hospital, 1959–1962
Clinical spectrum of individuals with de novo <scp><i>EBF3</i></scp> variants or deletions
Syndromic neurodevelopmental disorder associated with de novo variants in <scp><i>DDX23</i></scp>
Respiratory and otolaryngological disorders in Down syndrome from one center in Brazil
<scp>PAMI</scp> syndrome: A rare cause that can be easily misdiagnosed
<scp><i>HECW2</i></scp>‐related disorder in four Japanese patients Japanese
Delineating the <scp>Smith‐Kingsmore</scp> syndrome phenotype: Investigation of 16 patients with the <scp><i>MTOR</i></scp> c.<scp>5395G</scp> &gt; A p.(<scp>Glu1799Lys</scp>) missense variant
Elevated <scp>holo‐</scp>transcobalamin in Gaucher disease type <scp>II</scp>: A case report
Juvenile xanthogranuloma in Noonan syndrome
Phenotypically concordant but epigenetically discordant monozygotic dichorionic diamniotic twins with Beckwith–Wiedemann syndrome
An intrafamilial phenotypic variability in <scp>Ellis‐Van</scp> Creveld syndrome due to a novel 27 bps deletion mutation
Expanding the clinical spectrum in trichohepatoenteric syndrome
Expanding the genotypic spectrum of <scp><i>PYCR2</i></scp> and a common ancestry in Thai patients with hypomyelinating leukodystrophy 10
<scp><i>TSPEAR</i></scp> variants are primarily associated with ectodermal dysplasia and tooth agenesis but not hearing loss: A novel cohort study
New euchromatic variant dup(11)(p15.3p15.1) transmitted through two generations defined by low coverage whole genome sequencing
Blended phenotype of combination of <scp><i>HERC2</i></scp> and <scp><i>AP3B2</i></scp> deficiency and Angelman syndrome caused by paternal isodisomy of chromosome 15
Recurrence of an early postzygotic rescue of an inherited unbalanced translocation resulting in mosaic segmental uniparental isodisomy of chromosome 11q in siblings
<scp><i>CNOT2</i></scp> haploinsufficiency in a 40‐year‐old man with intellectual disability, autism, and seizures
An approach to rapid characterization of <scp><i>DMD</i></scp> copy number variants for prenatal risk assessment
Novel deep intronic and frameshift mutations causing a <scp><i>TRIP11</i></scp>‐related disorder
The contributions of careful clinical observations: A legacy
<scp>COXPD9</scp> in an individual from Puerto Rico and literature review
Cerebral folate transporter deficiency syndrome in three siblings: Why genetic testing for developmental and epileptic encephalopathies should be performed early and include the <scp><i>FOLR1</i></scp
Postoperative helmet therapy following fronto‐orbital advancement and cranial vault remodeling in patients with unilateral coronal synostosis
Phenotype‐driven variant filtration strategy in exome sequencing toward a high diagnostic yield and identification of 85 novel variants in 400 patients with rare Mendelian disorders
The delineation of the <scp>Wolf‐Hirschhorn</scp> syndrome over six decades: Illustration of the ongoing advances in phenotype analysis and cytogenomic technology
Simpson‐Golabi‐Behmel syndrome: One family, same mutation, different outcome
De novo variants in <scp><i>TCF7L2</i></scp> are associated with a syndromic neurodevelopmental disorder
A case of migraine treatment in a patient with a clinical diagnosis of <scp>CHARGE</scp> syndrome using onabotulinum toxin A
Mitochondrial cardiomyopathy and ventricular arrhythmias associated with biallelic variants in <scp><i>C1QBP</i></scp>
Genomic profiling in neuronal dyneinopathies and updated classifications
Cover Image, Volume 185A, Number 6, June 2021
Congenital chromoanagenesis in the routine postnatal chromosomal microarray analyses
Symptomatic mosaicism for a novel <scp><i>FBN1</i></scp> splice site variant in a parent causing inherited neonatal Marfan syndrome
New Framework Developed for Polygenic Risk Score Reporting
Table of Contents, Volume 185A, Number 6, June 2021
Conference Attendees Give Thumbs up to Virtual Meetings
Skeletal and molecular findings in 51 Cleidocranial dysplasia patients from Turkey
Publication schedule for 2021
A family with partially penetrant multicentric carpotarsal osteolysis due to gonadal mosaicism: First reported case
In This Issue
Heritable disorders of oxygen sensing
<scp>Nonclassic</scp> fibrodysplasia ossificans progressiva: A child from Angola with an <scp>ACVR1<sup>G328E</sup></scp> variant
Recurrent <scp><i>NFIA</i> K125E</scp> substitution represents a loss‐of‐function allele: Sensitive in vitro and in vivo assays for nontruncating alleles
Two novel bi‐allelic <scp><i>KDELR2</i></scp> missense variants cause osteogenesis imperfecta with neurodevelopmental features
The genotypic and phenotypic spectrum of pycnodysostosis in Saudi Arabia: Novel variants and clinical findings
Positive response to imatinib in <scp><i>PDGFRB</i></scp>‐related Kosaki overgrowth syndrome
Trisomy 9 mosaic syndrome: Sixteen additional patients with new and/or less commonly reported features, literature review, and suggested clinical guidelines
<scp><i>SATB2</i>‐associated</scp> syndrome in adolescents and adults
Cardiac abnormalities in girls with Turner syndrome: <scp>ECG</scp> abnormalities, myocardial strain imaging, and karyotype–phenotype associations
Next‐generation sequencing and the evolution of data sharing
Evaluation of sleep‐disordered breathing and its relationship with respiratory parameters in children with mucopolysaccharidosis Type <scp>IVA</scp> and <scp>VI</scp>
Congenital defects of glycosylation: Novel presentations with mainly neurological involvement and variable dysmorphic features
A new syndrome of moyamoya disease, kidney dysplasia, aminotransferase elevation, and skin disease associated with de novo variants in <scp><i>RNF213</i></scp>
Microdeletion of 9q22.3: A patient with minimal deletion size associated with a severe phenotype
Death rates in the U.S. due to Leukodystrophies with pediatric forms
Lower birth weight in newborns with trisomy 18 and esophageal atresia
Pediatric Cushing syndrome: An early sign of an underling cancer predisposition syndrome
Classifications of split hand foot malformation (SHFM) should include transverse deficiencies: Why Maisels was correct
Reflections on the history of genetic medicine at Johns Hopkins University
From cataract to syndrome diagnosis: Revaluation of <scp>Warburg‐Micro</scp> syndrome <scp>Type</scp> 1 patients
Fifty years of recognizable patterns of human malformation: Insights and opportunities
<scp>Natural history study of adults with Wolf–Hirschhorn syndrome</scp> 2: Patient‐reported outcomes study
A novel de novo intronic variant in <scp><i>ITPR1</i></scp> causes Gillespie syndrome
Novel <scp><i>GUCY2C</i></scp> variant causing familial diarrhea in a Mennonite kindred and a potential therapeutic approach Mennonite
Genotype–phenotype correlation in <scp>Phelan‐McDermid</scp> syndrome: A comprehensive review of chromosome 22q13 deleted genes
Childhood onset nexilin dilated cardiomyopathy: A heterozygous and a homozygous case
Natural history of alpha‐thalassemia X‐linked intellectual disability syndrome: A case report of a 45‐year‐old man
Genetic heterogeneity of disorders with overgrowth and intellectual disability: Experience from a center in North India
Biallelic <scp><i>ASCC1</i></scp> variants including a novel intronic variant result in expanded phenotypic spectrum of spinal muscular atrophy with congenital bone fractures 2 (<scp>SMABF2</scp>)
A de novo missense variant in <scp><i>MED13</i></scp> in a patient with global developmental delay, marked facial dysmorphism, macroglossia, short stature, and macrocephaly
The Dysmorphology Unit from 1976 to 1980: Fleeting fellow, deformations, and John Graham
Beyond nephronophthisis: Retinal dystrophy in the absence of kidney dysfunction in childhood expands the clinical spectrum of <scp>CEP83</scp> deficiency
Craniofacial features of 3q29 deletion syndrome: Application of next‐generation phenotyping technology
Hypoxia: A teratogen underlying a range of congenital disruptions, dysplasias, and malformations
Sleep‐disordered breathing and its management in children with rare skeletal dysplasias
Two females with distinct de novo missense pathogenic variants in <scp><i>MED12</i></scp> and vastly differing phenotypes
Identification of a novel pathogenic variant in <scp><i>CKAP2L</i></scp> and literature review in a child with Filippi syndrome and congenital talipes equinovarus
Thinking outside “The Box”: <scp>Case‐based</scp> didactics for medical education and the instructional legacy of Dr John M. Graham, Jr
Spread of X‐chromosome inactivation into autosomal regions in patients with unbalanced X‐autosome translocations and its phenotypic effects
Characterization of sleep habits of children with Sotos syndrome
Co‐occurring non‐omphalocele and non‐gastroschisis anomalies among cases with congenital omphalocele and gastroschisis
Heterozygous missense variant in <scp><i>TRPC6</i></scp> in a boy with rapidly progressive infantile nephrotic syndrome associated with diffuse mesangial sclerosis
Novel <scp><i>MYT1</i></scp> variants expose the complexity of oculo‐auriculo‐vertebral spectrum genetic mechanisms
A journey towards answers: Bonnie Odgers Meets Dr. John Graham
Marfan syndrome resulting from a rare pathogenic <scp><i>FBN1</i></scp> variant, ascertained through a proband with <scp>IgG4</scp>‐related arteriopathy
Hospital care of patients with inherited cardiomyopathies in Germany during the Covid‐19 pandemic insights from the German‐wide Helios hospital network
A lasting imprint
Variants associated with urea cycle disorders in Japanese patients: Nationwide study and literature review
Hypotrichosis‐lymphedema‐telangiectasia syndrome: Report of ileal atresia associated with a <scp><i>SOX18</i></scp> de novo pathogenic variant and review of the phenotypic spectrum
Heterozygous variants in <i>SPTBN1</i> cause intellectual disability and autism
Parental mosaicism in de novo neurodevelopmental diseases
To John M. Graham Jr, who called me into a career in clinical genetics
Craniosynostosis is a feature of <scp><i>CHD7</i></scp>‐related <scp>CHARGE</scp> syndrome
Resolution of sclerotic lesions of dysosteosclerosis due to biallelic <i>SLC29A3</i> variant in a Turkish girl
Choose your words carefully
Glutamine Linked to Cell Senescence and Aging
In This Issue
Hyperbaric oxygen management of recurrent cellulitis in poikiloderma with neutropenia
Publication schedule for 2021
Table of Contents, Volume 185A, Number 5, May 2021
Identical Twins not as “Identical” as Previously Thought
Cover Image, Volume 185A, Number 5, May 2021
Subtle differences in autonomic symptoms in people diagnosed with hypermobile Ehlers–Danlos syndrome and hypermobility spectrum disorders
<i>SLC35F1</i> as a candidate gene for neurodevelopmental disorders resembling Rett syndrome
<scp>Smith–Magenis</scp> syndrome: Report of morphological and new functional cardiac findings with review of the literature
Feingold syndrome type 2 in a patient from China Chinese population
A novel homozygous <scp><i>SLC13A5</i></scp> whole‐gene deletion generated by <scp><i>Alu/Alu</i></scp>‐mediated rearrangement in an Iraqi family with epileptic encephalopathy Iraqi family; distinct population
Expanding the genotypic and phenotypic spectrum in a diverse cohort of 104 individuals with Wiedemann‐Steiner syndrome
Further delineation of van den <scp>Ende‐Gupta</scp> syndrome: Genetic heterogeneity and overlap with congenital heart defects and skeletal malformations syndrome
A novel <scp><i>EZH2</i></scp> gene variant in a case of Weaver syndrome with postaxial polydactyly
Recurrent ganglioneuroma in <scp><i>PTPN11</i></scp>‐associated Noonan syndrome: A case report and literature review
<scp>The mystery of monozygotic twinning II</scp>: What can monozygotic twinning tell us about Amyoplasia from a review of the various mechanisms and types of monozygotic twinning?
<scp><i>MASP1</i></scp>‐related <scp>3MC</scp> syndrome in a patient from Turkey Kurdish origin
Factors that increase risk for poor adherence to phenylketonuria treatment in Brazilian patients
Genotype and phenotype in 18 Chinese patients with <scp>Coffin‐Siris</scp> syndrome Chinese; ethnicity-related effect (phrases: 'Chinese individuals', 'Chinese patients', 'ethnicity-re
Clinical and molecular characterization of patients with hereditary hemorrhagic telangiectasia: Experience from an <scp>HHT</scp> Center of Excellence
A rare case of postnatal mosaic trisomy 12 with severe congenital heart disease and literature review
<scp>The mystery of monozygotic twinning</scp> I: What can Amyoplasia tell us about monozygotic twinning and the possible role of <scp>twin–twin</scp> transfusion?
Natural history study of adults with <scp>Wolf–Hirschhorn</scp> syndrome 1: Case series of personally observed 35 individuals
<scp><i>MYH1</i></scp> is a candidate gene for recurrent rhabdomyolysis in humans
Pathogenic variants in <scp><i>KCNQ2</i></scp> cause intellectual deficiency without epilepsy: Broadening the phenotypic spectrum of a potassium channelopathy
Advances in genomic diagnosis of a large cohort of Egyptian patients with disorders of sex development
First prenatal case of <scp>Noonan</scp> syndrome with <scp><i>SOS2</i></scp> mutation: Implications of early diagnosis for genetic counseling
Report of a novel variant in the <scp><i>FAM111A</i></scp> gene in a fetus with multiple anomalies including gracile bones, hypoplastic spleen, and hypomineralized skull
Confined placental mosaicism involving multiple de novo copy number variants associated with fetal growth restriction: A case report
Hedgehog acyl‐transferase‐related multiple congenital anomalies: Report of an additional family and delineation of the syndrome
Patterns of congenital anomalies among individuals with trisomy 13 in Texas
Further expanding the mutational spectrum of <scp>brain abnormalities, neurodegeneration, and dysosteosclerosis</scp>: A rare disorder with neurologic regression and skeletal features
<scp><i>R3HDM1</i></scp> haploinsufficiency is associated with mild intellectual disability
Hemimegalencephaly and intractable seizures associated with the <i>NPRL3</i> gene variant in a newborn: A case report
Parent‐reported histories of adults with trisomy 13 syndrome
A case of <scp>Ververi‐Brady</scp> syndrome due to <scp><i>QRICH1</i></scp> loss of function and the literature review
Specialty clinics for adults with Down syndrome: A clinic survey
A novel <scp><i>MPLKIP</i></scp>‐variant in three Finnish patients with non‐photosensitive trichothiodystrophy type 4 Finnish
Cobalamin J disease detected on newborn screening: Novel variant and normal neurodevelopmental course
Reflections on a career in dysmorphology, teratology, and clinical genetics
A novel homozygous <scp><i>RIPK4</i></scp> variant in a family with severe <scp>Bartsocas‐Papas</scp> syndrome
California Continues to Support Stem Cell Research
A New “Consensus Genome” Significantly Improves the Accuracy of RNA‐Seq Analyses
Table of Contents, Volume 185A, Number 4, April 2021
Publication schedule for 2021
Cover Image, Volume 185A, Number 4, April 2021
In This Issue
A novel microduplication in <scp><i>INPP5A</i></scp> segregates with schizophrenia spectrum disorder in the family of a patient with both childhood onset schizophrenia and autism spectrum disorder
Genetic and metabolic investigations for individuals with neurodevelopmental disorders: A survey of Canadian geneticists' practices
Surveillance guidelines for children with trisomy 13
The first adolescent case of Fraser syndrome 3, with a novel nonsense variant in <i>GRIP1</i>
A novel <scp><i>ALG14</i></scp> missense variant in an alive child with myopathy, epilepsy, and progressive cerebral atrophy
Understanding the phenotypic spectrum of <scp><i>ASXL</i></scp>‐related disease: Ten cases and a review of the literature
Clinical and genetic characterization of <scp><i>PYROXD1</i></scp>‐related myopathy patients from Turkey Turkish; patients from Turkey; Turkish founder mutation; Turkish families
An adult Chinese patient with developmental delay with short stature, dysmorphic features, and sparse hair (<scp>Loucks‐Innes</scp> syndrome)
Investigation of (epi)genotype causes and follow‐up manifestations in the patients with classical and atypical phenotype of<scp>Beckwith‐Wiedemann</scp>spectrum
Management of <scp>COVID</scp>‐19 infection in organic acidemias
Expanding the clinical phenotype of the ultra‐rare <scp>Skraban‐Deardorff</scp> syndrome: Two novel individuals with <scp><i>WDR26</i></scp> loss‐of‐function variants and a literature review
Segmental overgrowth and aneurysms due to mosaic <scp><i>PDGFRB</i></scp> p.(<scp>Tyr562Cys</scp>)
<scp>Aicardi‐Goutières</scp> syndrome may present with positive newborn screen for X‐linked adrenoleukodystrophy
Congenital polyvalvular disease expands the cardiac phenotype of the <scp>RASopathies</scp>
Living history biography: An afterthought
Severe epileptic encephalopathy associated with compound heterozygosity of <scp>THG1L</scp> variants in the Ashkenazi Jewish population Ashkenazi Jewish
Trends in the prenatal diagnosis of trisomy 21 show younger maternal age and shift in the distribution of congenital heart disease over a 20‐year period
Deformations associated with arthrogryposis
The society for craniofacial genetics and developmental biology 43rd annual meeting
<scp>Birt‐Hogg‐Dubé</scp> symptoms in <scp>Smith‐Magenis</scp> syndrome include pediatric‐onset pneumothorax
Partners in care
Sleep and behavior in children and adolescents with tuberous sclerosis complex
Presacral neuroendocrine tumors associated with the Currarino syndrome
Establishing intellectual disability as the key feature of patients with biallelic <scp><i>RNPC3</i></scp> variants
<scp>ELOVL4</scp> with erythrokeratoderma: A pediatric case and emerging genodermatosis
Childhood prevalence of achondroplasia in New South Wales and the Australian Capital Territory, Australia
Novel <scp><i>USP9X</i></scp> variant associated with syndromic intellectual disability in a female: A case study and review
Novel unconventional variants expand the allelic spectrum of <scp><i>OPHN1</i></scp> gene
Cerebellofaciodental syndrome in an adult patient: Expanding the phenotypic and natural history characteristics
Coexistence of severe developmental delay, epilepsy, and hemangioma in Snijders <scp>Blok‐Fisher</scp> syndrome suggests the presence of a <scp><i>POU3F3</i></scp>‐related <scp>SNIBFIS</scp> endopheno
3137 fetuses in 33 years: What we have learned from the Wisconsin stillbirth service program
Medulloblastoma in the setting of megalencephaly polymicrogyria polydactyly hydrocephalus
14q32.11 microdeletion including <scp><i>CALM1</i></scp>, <scp><i>TTC7B</i></scp>, <scp><i>PSMC1</i></scp>, and <scp><i>RPS6KA5</i></scp>: A new potential cause of developmental and language delay in
Whole genome sequencing of 45 Japanese patients with intellectual disability Japanese
The phenotype of 15 cases with rare 8q24.13‐q24.3 deletions–A new syndrome or still an enigma?
Fork‐shaped mandibular incisors as a novel phenotype of <scp>LRP5</scp>‐associated disorder
X‐linked frontometaphyseal dysplasia with severe scoliosis and spinal cord compromise in an Indian boy
Case report of adrenocortical carcinoma associated with double germline mutations in <scp><i>MSH2</i></scp> and <scp><i>RET</i></scp>
Clinical phenotype and management of individuals with mosaic monosomy X with Y chromosome material stratified by genital phenotype
Direct visualization of the evolution of limb amputation in amnion rupture sequence in an extremely preterm infant born at 22 weeks
Temple syndrome resulting from uniparental disomy is undiagnosed by a methylation assay due to low‐level mosaicism for trisomy 14
Further delineation of <scp><i>SMG9</i></scp>‐related heart and brain malformation syndrome
Language in young females with fragile X syndrome: Influence on the neurocognitive profile and adaptive behavior
Everolimus for severe arrhythmias in tuberous sclerosis complex related cardiac rhabdomyomas
A founder mutation in <scp><i>TCTN2</i></scp> causes <scp>Meckel‐Gruber</scp> syndrome type 8 among Jews of Ethiopian and Yemenite origin Jews of Ethiopian and Yemenite origin
Parent‐authored memoirs: Lessons in the practice of narrative medicine
Maternal health and pregnancy outcome in diagnosed and undiagnosed Marfan syndrome: A registry‐based study
A novel variant in <scp><i>YWHAG</i></scp> further supports phenotype of developmental and epileptic encephalopathy
Twins with <scp>PEX7</scp> related intellectual disability and cataract: Highlighting phenotypes of peroxisome biogenesis disorder <scp>9B</scp>
A phenotypically diverse family with an atypical 22q11.2 deletion due to an unbalanced 18q23;22q11.2 translocation
Germ cell mosaicism for <scp><i>AUTS2</i></scp> exon 6 deletion
Clinical characterization of individuals with the distal 1q21.1 microdeletion
Alazami syndrome: Report of three Indian patients with phenotypic spectrum from adolescence to adulthood Indian; Saudi Arabian
Manifestations of thrombospondin type‐1 domain‐containing protein 1 gene mutation in an extremely premature infant with nonimmune hydrops fetalis
Gene Editing has Potential as Treatment for Progeria
In This Issue
Cover Image, Volume 185A, Number 3, March 2021
Table of Contents, Volume 185A, Number 3, March 2021
Florida Passes Genetic Non‐Discrimination Law
Wiedemann–Rautenstrauch syndrome in an Indian patient with biallelic pathogenic variants in <scp><i>POLR3A</i></scp> Indian
Publication schedule for 2021
Characterizing upper limb function in the context of activities of daily living in <scp>CLN3</scp> disease
Nager syndrome in patient lacking acrofacial dysostosis: Expanding the phenotypic spectrum of <scp><i>SF3B4</i></scp>‐related disease
A dihydrofolate reductase 2 (<i><scp>DHFR2</scp>)</i> variant is associated with risk of neural tube defects in an Irish cohort but not in a United Kingdom cohort
A novel heterotaxy gene: Expansion of the phenotype of <scp><i>TTC21B</i>‐spectrum</scp> disease
Expansion of the ophthalmic phenotype of <scp><i>SPINT2</i></scp>‐related syndromic congenital sodium diarrhea
Fetal early motor neuron disruption and prenatal molecular diagnosis in a severe <scp>BICD2</scp>‐opathy
<scp>The point‐of‐care</scp> use of a facial phenotyping tool in the genetics clinic: Enhancing diagnosis and education with machine learning
Neurodevelopmental disorder in an Egyptian family with a biallelic <scp><i>ALKBH8</i></scp> variant Egyptian descent; Saudi Arabian kindreds; distinct population
Fragile X syndrome full mutation in cognitively normal male identified as part of an Australian reproductive carrier screening program
Long‐term weight control in adults with <scp>Prader‐Willi</scp> syndrome living in residential hostels
Auditory phenotype of <scp>Smith–Lemli–Opitz</scp> syndrome
Ophthalmic phenotypes associated with biallelic loss‐of‐function <scp><i>PCDH12</i></scp> variants
Surveillance guidelines for children with trisomy 18
Phenotypic expansion of the <scp><i>BPTF</i></scp>‐related neurodevelopmental disorder with dysmorphic facies and distal limb anomalies
Early prenatal diagnosis of alveolar capillary dysplasia with misalignment of pulmonary veins due to a 16q24.1 deletion
Common pathogenesis for sirenomelia, <scp>OEIS</scp> complex, <scp>limb‐body</scp> wall defect, and other malformations of caudal structures
Variants in <scp><i>KIF2A</i></scp> cause broad clinical presentation; the computational structural analysis of a novel variant in a patient with a cortical dysplasia, complex, with other brain malfor
Genetic skin disorders: The value of a multidisciplinary clinic
<scp>CLARITY</scp>: Co‐occurrences in achondroplasia—craniosynostosis, seizures, and decreased risk of diabetes mellitus
Clinical and molecular characterization of four patients with Robinow syndrome from different families Indian families
Circulating free <scp>DNA</scp> in the plasma of individuals with neurofibromatosis type 1
Noonan syndrome with loose anagen hair with variants in the <scp><i>PPP1CB</i></scp> gene: First familial case reported
Novel genetic testing model: A collaboration between genetic counselors and nephrology
Sleep phenotype of individuals with autism spectrum disorder bearing mutations in the <scp><i>PER2</i></scp> circadian rhythm gene
Novel <scp><i>FOXP1</i></scp> pathogenic variants in two Indian subjects with syndromic intellectual disability Indian
Lessons from a 30 year follow‐up of monozygotic twins with discordant phenotype due to a ring 13 chromosomal mosaicism in one of them
Liver transplant as a curative treatment in a pediatric patient with classic homocystinuria: A case report
Craniofacial and occlusal features of children with Noonan syndrome
Aldosterone synthase (<scp>CYP11B2</scp>) deficiency among Palestinian infants: Three novel variants and genetic heterogeneity Palestinian; Arab
Human disease genes website series: An international, open and dynamic library for up‐to‐date clinical information
Evidence that <scp><i>FGFRL1</i></scp> contributes to congenital diaphragmatic hernia development in humans
Interfamilial clinical variability in four <scp>Polish</scp> families with cranioectodermal dysplasia and identical compound heterozygous variants in <scp><i>WDR35</i></scp> Polish
<scp><i>AIFM1</i></scp>‐associated X‐linked spondylometaphyseal dysplasia with cerebral hypomyelination
First reported adult patient with retinal dystrophy and leukodystrophy caused by a novel <scp>ACBD5</scp> variant: A case report and review of literature
Hyperinsulinism in an individual with an <scp><i>EP300</i></scp> variant of <scp>Rubinstein‐Taybi</scp> syndrome
41st Annual David W. Smith workshop on malformations and morphogenesis: Abstracts of the 2020 annual meeting
Intellectual disability associated with craniofacial dysmorphism, cleft palate, and congenital heart defect due to a de novo <scp><i>MEIS2</i></scp> mutation: A clinical longitudinal study
Table of Contents, Volume 185A, Number 2, February 2021
In This Issue
<scp><i>MBD5‐</i></scp>related intellectual disability in a Vietnamese child Vietnamese
Clinical and molecular characterizations of 11 new patients with type 1 Feingold syndrome: Proposal for selecting diagnostic criteria and further genetic testing in patients with severe phenotype
Publication schedule for 2021
Genome Research Institute Identifies Vision for the Future
<i>Gpr12</i> Linked to Short‐Term Memory
Cover Image, Volume 185A, Number 2, February 2021
Can Waardenburg syndrome type 2 be explained by epigenetic mosaicism?
Cancer surveillance in children with Ollier Disease and Maffucci Syndrome
Adult diagnosis of <scp>Townes–Brocks</scp> syndrome with renal failure: Two related cases and review of literature
Genotype–phenotype correlation in <scp><i>GNB1</i></scp>‐related neurodevelopmental disorder: Potential association of p.<scp>Leu95Pro</scp> with cleft palate
Expansion of <scp><i>NEUROD2</i></scp> phenotypes to include developmental delay without seizures
Phenotypic spectrum associated with pathogenic mutation in the <scp>NRG1</scp> gene in Acadian family Acadian descent
<scp><i>SCYL1</i></scp> disease and liver transplantation diagnosed by reanalysis of exome sequencing and deletion/duplication analysis of <scp><i>SCYL1</i></scp>
Clinical diagnosis of neurofibromatosis type I in multiple family members due to cosegregation of a unique balanced translocation with disruption of the <scp><i>NF1</i></scp> locus: Testing considerat
Neurocognitive assessment and DNA sequencing expand the phenotype and genotype spectrum of Alström syndrome
A validated model for prediction of survival to 6 months in patients with trisomy 13 and 18
<scp>SRD5A3‐CDG</scp>:<scp>3D</scp>structure modeling, clinical spectrum, and<scp>computer‐based</scp>dysmorphic facial recognition