European Journal of Human Genetics - 2018

273 articles | Last updated: 2025-12-03 14:12:56
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Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?
Recontacting clinical genetics patients with reclassified results: equity and policy challenges
Diaspora, migration, and the sciences: a new integrated perspective
Bone morphogenetic protein 4 (BMP4) loss-of-function variant associated with autosomal dominant Stickler syndrome and renal dysplasia
A tyrosine kinase-activating variant Asn666Ser in PDGFRB causes a progeria-like condition in the severe end of Penttinen syndrome
Choosing not to know: accounts of non-engagement with pre-symptomatic testing for Machado-Joseph disease
A game of hide and seq: Identification of parallel Y-STR evolution in deep-rooting pedigrees
Communication about genetic testing with breast and ovarian cancer patients: a scoping review
Skewed X-inactivation is common in the general female population
A National French consensus on gene lists for the diagnosis of myopathies using next-generation sequencing
The role of recessive inheritance in early-onset epileptic encephalopathies: a combined whole-exome sequencing and copy number study
Holt-Oram syndrome: clinical and molecular description of 78 patients with TBX5 variants
De novo truncating variants in PHF21A cause intellectual disability and craniofacial anomalies
The role of matrilineality in shaping patterns of Y chromosome and mtDNA sequence variation in southwestern Angola
A rare regulatory variant in the MEF2D gene affects gene regulation and splicing and is associated with a SLE sub-phenotype in Swedish cohorts Swedish cohorts
Diagnostics of short tandem repeat expansion variants using massively parallel sequencing and componential tools
Assessment of fibroblast nuclear morphology aids interpretation of LMNA variants
Mendelian randomization reveals unexpected effects of CETP on the lipoprotein profile
Genetic variation in the Estonian population: pharmacogenomics study of adverse drug effects using electronic health records
Comparative perspectives: regulating insurer use of genetic information
Deletion in 2q35 excluding the IHH gene leads to fetal severe limb anomalies and suggests a disruption of chromatin architecture
Back to the drawing board—loss of chromosome Y (LOY) in leukocytes is associated with age-related macular degeneration
A stroke gene panel for whole-exome sequencing
Genetic Counselling for the Laboratory: building bridges between disciplines
TBL1Y: a new gene involved in syndromic hearing loss
The Medical Genome Reference Bank: a whole-genome data resource of 4000 healthy elderly individuals. Rationale and cohort design
Mitochondrial complex IV deficiency caused by a novel frameshift variant in MT-CO2 associated with myopathy and perturbed acylcarnitine profile
Functional reassessment of PAX6 single nucleotide variants by in vitro splicing assay
Recontacting patients in clinical genetics services: recommendations of the European Society of Human Genetics
The comprehensive mutational and phenotypic spectrum of TUBB8 in female infertility
Offering a choice between NIPT and invasive PND in prenatal genetic counseling: the impact of clinician characteristics on patients’ test uptake
Presymptomatic testing of those at 25% risk of autosomal dominant neurodegenerative disease- testing team beware
Heterozygous loss-of-function variants of MEIS2 cause a triad of palatal defects, congenital heart defects, and intellectual disability
1 in 38 individuals at risk of a dominant medically actionable disease
A new case of SMA phenotype without epilepsy due to biallelic variants in ASAH1
The Global State of the Genetic Counseling Profession
Presymptomatic genetic testing for hereditary cancer in young adults: a survey of young adults and parents
Newborn dried blood spot samples in Denmark: the hidden figures of secondary use and research participation
Abstracts from the 50th European Society of Human Genetics Conference: Program
Abstracts from the 50th European Society of Human Genetics Conference: Oral Presentations
Pilot study of expanded carrier screening for 11 recessive diseases in China: results from 10,476 ethnically diverse couples
European guidelines for constitutional cytogenomic analysis European guidelines for constitutional cytogenomic analysis
Abstracts from the 50th European Society of Human Genetics Conference: Posters
Abstracts from the 50th European Society of Human Genetics Conference: Electronic Posters
Patients v. Myriad or the GDPR Access Right v. the EU Database Right
Heterozygous LHX3 mutations may lead to a mild phenotype of combined pituitary hormone deficiency
Combined linkage and association analysis identifies rare and low frequency variants for blood pressure at 1q31
Novel calcineurin A (PPP3CA) variant associated with epilepsy, constitutive enzyme activation and downregulation of protein expression
The recognition of the profession of Genetic Counsellors in Europe
Systems genetics of nonsyndromic orofacial clefting provides insights into its complex aetiology
Novel insight into the genetic basis of high-altitude pulmonary hypertension in Kyrgyz highlanders
Fetal fraction evaluation in non-invasive prenatal screening (NIPS)
A weighted burden test using logistic regression for integrated analysis of sequence variants, copy number variants and polygenic risk score
Variant in SCYL1 gene causes aberrant splicing in a family with cerebellar ataxia, recurrent episodes of liver failure, and growth retardation
Centenarian controls increase variant effect sizes by an average twofold in an extreme case–extreme control analysis of Alzheimer’s disease
Stereocilin gene variants associated with episodic vertigo: expansion of the DFNB16 phenotype
Search for cis-acting factors and maternal effect variants in Silver-Russell patients with ICR1 hypomethylation and their mothers
Meta-analysis of genome-wide association studies of aggressive and chronic periodontitis identifies two novel risk loci
Genetic testing for DADA2: How can we avoid missing patients?
MultiWaver 2.0: modeling discrete and continuous gene flow to reconstruct complex population admixtures
Reply to Sönmez et al.
Reverse pre-symptomatic testing for Huntington disease: double disclosure when 25% at-risk children reveal the genetic status to their parent
Psychosocial and behavioral outcomes of genomic testing in cancer: a systematic review
A genome-wide search for new imprinted genes in the human placenta identifies DSCAM as the first imprinted gene on chromosome 21
Exploring rare and low-frequency variants in the Saguenay–Lac-Saint-Jean population identified genes associated with asthma and allergy traits
Exploring predictive biomarkers from clinical genome-wide association studies via multidimensional hierarchical mixture models
Detection and quantification of a KIF11 mosaicism in a subject presenting familial exudative vitreoretinopathy with microcephaly
Next-generation sequencing identified SPATC1L as a possible candidate gene for both early-onset and age-related hearing loss
A fascinating overview of the biology of fragile X syndrome
Y chromosome mosaicism is associated with age-related macular degeneration
Analysis of VUS reporting, variant reinterpretation and recontact policies in clinical genomic sequencing consent forms
Clinical utility gene card for: inherited optic neuropathies including next-generation sequencing-based approaches
The radial expansion of the Diego blood group system polymorphisms in Asia: mark of co-migration with the Mongol conquests
Genetics of hearing loss in the Arab population of Northern Israel
DNAJC12-associated developmental delay, movement disorder, and mild hyperphenylalaninemia identified by whole-exome sequencing re-analysis
Correction: Evaluation of copy-number variants as modifiers of breast and ovarian cancer risk for BRCA1 pathogenic variant carriers
New splicing pathogenic variant in EBP causing extreme familial variability of Conradi–Hünermann–Happle Syndrome
Linear isoforms of the long noncoding RNA CDKN2B-AS1 regulate the c-myc-enhancer binding factor RBMS1
A study of Kibbutzim in Israel reveals risk factors for cardiometabolic traits and subtle population structure
Characterization of three ciliopathy pedigrees expands the phenotype associated with biallelic C2CD3 variants
On the loss of human sex chromosomes in lymphocytes with age: a quantitative treatment
A patient-specific induced pluripotent stem cell model for West syndrome caused by ST3GAL3 deficiency
Estimating the age of p.(Phe508del) with family studies of geographically distinct European populations and the early spread of cystic fibrosis European populations
Guidelines for reporting secondary findings of genome sequencing in cancer genes: the SFMPP recommendations
Current practices for access, compensation, and prioritization in biobanks. Results from an interview study
Application of the parametric bootstrap for gene-set analysis of gene–environment interactions
Genetic variation in CHRNA7 and CHRFAM7A is associated with nicotine dependence and response to varenicline treatment
Primary congenital glaucoma including next-generation sequencing-based approaches: clinical utility gene card
CUGC for pontocerebellar hypoplasia type 9 and spastic paraplegia-63
Sequence diversity of the Rh blood group system in Basques
Biallelic loss of function variants in COASY cause prenatal onset pontocerebellar hypoplasia, microcephaly, and arthrogryposis
Functional characterization of common BCL11B gene desert variants suggests a lymphocyte-mediated association of BCL11B with aortic stiffness
Association of modifiers and other genetic factors explain Marfan syndrome clinical variability
An MTF1 binding site disrupted by a homozygous variant in the promoter of ATP7B likely causes Wilson Disease
Registered access: authorizing data access
Expanding the phenotypic spectrum of variants in PDE4D/PRKAR1A: from acrodysostosis to acroscyphodysplasia
Human urine-derived renal epithelial cells provide insights into kidney-specific alternate splicing variants
Before progressing from “exomes” to “genomes”… don’t forget splicing variants
Paternal origin of Paleo-Indians in Siberia: insights from Y-chromosome sequences
Lack of evidence for a causal role of CALR3 in monogenic cardiomyopathy
Molecular genetic overlap between migraine and major depressive disorder
Inferring biogeographic ancestry with compound markers of slow and fast evolving polymorphisms
Could the interaction between LMX1B and PAX2 influence the severity of renal symptoms?
Correction: Patient experience and utility of genetic information: a cross-sectional study among patients tested for cancer susceptibility and thrombophilia
Incidental or secondary findings: an integrative and patient-inclusive approach to the current debate
Multi-level genomic analyses suggest new genetic variants involved in human memory
Comparison of methods for transcriptome imputation through application to two common complex diseases
Genome-wide copy number variation analysis identifies novel candidate loci associated with pediatric obesity
Genetic diversity of NDUFV1-dependent mitochondrial complex I deficiency
Pharmacogenomics, a novel section in the European Journal of Human Genetics
De novo repeat interruptions are associated with reduced somatic instability and mild or absent clinical features in myotonic dystrophy type 1
A fine-mapping study of central obesity loci incorporating functional annotation and imputation
Optimization of the diagnosis of inherited colorectal cancer using NGS and capture of exonic and intronic sequences of panel genes
Whole-exome sequencing in intellectual disability; cost before and after a diagnosis
Primary brain calcification: an international study reporting novel variants and associated phenotypes
Project MinE: study design and pilot analyses of a large-scale whole-genome sequencing study in amyotrophic lateral sclerosis
Phenotype-loci associations in networks of patients with rare disorders: application to assist in the diagnosis of novel clinical cases
De novo ITPR1 variants are a recurrent cause of early-onset ataxia, acting via loss of channel function
A Faroese founder variant in TBCD causes early onset, progressive encephalopathy with a homogenous clinical course
SET de novo frameshift variants associated with developmental delay and intellectual disabilities
Differences in attitudes toward genetic testing among the public, patients, and health-care professionals in Korea
Isochromosome 21q is overrepresented among false-negative cell-free DNA prenatal screening results involving Down syndrome
Developmental epileptic encephalopathy with hypomyelination and brain atrophy associated with PTPN23 variants affecting the assembly of UsnRNPs
Knockdown of Crispld2 in zebrafish identifies a novel network for nonsyndromic cleft lip with or without cleft palate candidate genes
A heterozygous microdeletion of 20q13.13 encompassing ADNP gene in a child with Helsmoortel–van der Aa syndrome
Utility of two SMN1 variants to improve spinal muscular atrophy carrier diagnosis and genetic counselling
Correction: Cost of cancer diagnosis using next-generation sequencing targeted gene panels in routine practice: a nationwide French study
Careful what you say
Cancer prevention by aspirin in children with Constitutional Mismatch Repair Deficiency (CMMRD)
Phenotypic spectrum of Au–Kline syndrome: a report of six new cases and review of the literature
A theory-informed systematic review of clinicians’ genetic testing practices
Expanding the clinical and molecular findings in RASA1 capillary malformation-arteriovenous malformation
Broad phenotypes in heterozygous NR5A1 46,XY patients with a disorder of sex development: an oligogenic origin?
A three-generation family with metaphyseal dysplasia, maxillary hypoplasia and brachydactyly (MDMHB) due to intragenic RUNX2 duplication
Trans-eQTLs identified in whole blood have limited influence on complex disease biology
Nephronectin (NPNT) and the prediction of nephrotic syndrome response to steroid treatment
Biallelic variants in the ciliary gene TMEM67 cause RHYNS syndrome
Uniparental isodisomy as a cause of recessive Mendelian disease: a diagnostic pitfall with a quick and easy solution in medium/large NGS analyses
How are genetic test results being used by Australian life insurers?
Genetically driven adiposity traits increase the risk of coronary artery disease independent of blood pressure, dyslipidaemia, glycaemic traits
The phenotypic spectrum of proximal 6q deletions based on a large cohort derived from social media and literature reports
Rett syndrome: a seminal book on extensive multidisciplinary analyses for rare disease
Patient preferences for massively parallel sequencing genetic testing of colorectal cancer risk: a discrete choice experiment
Frequency of genetic variants associated with arrhythmogenic right ventricular cardiomyopathy in the genome aggregation database
Reply to “Between SCA5 and SCAR14: delineation of the SPTBN2 p.R480W-associated phenotype” by Nuovo et al.
More than an information service: are counselling skills needed by genetics professionals in the genomic era?
Between SCA5 and SCAR14: delineation of the SPTBN2 p.R480W-associated phenotype
A recurrent de novo CUX2 missense variant associated with intellectual disability, seizures, and autism spectrum disorder
Economic evaluation of genomic sequencing in the paediatric population: a critical review
The FAIR guiding principles for data stewardship: fair enough?
CUGC for congenital primary aphakia
Clinical and functional characterization of the CDH1 germline variant c.1679C>G in three unrelated families with hereditary diffuse gastric cancer
Risk of multiple pancreatic cancers in CDKN2A-p16-Leiden mutation carriers
Clinical progression and metachronous paragangliomas in a large cohort of SDHD germline variant carriers
Microcephaly, short stature, and limb abnormality disorder due to novel autosomal biallelic DONSON mutations in two German siblings
Evidence that UBASH3 is a causal gene for type 1 diabetes
Intentions to share exome sequencing results with family members: exploring spousal beliefs and attitudes
A novel NAA10 variant with impaired acetyltransferase activity causes developmental delay, intellectual disability, and hypertrophic cardiomyopathy
Circulating microRNAs disclose biology of normal cognitive function in healthy elderly people – a discovery twin study
Whole-genome sequencing reveals new insights into age-related hearing loss: cumulative effects, pleiotropy and the role of selection
The Genomics ADvISER: development and usability testing of a decision aid for the selection of incidental sequencing results
Comprehensive genomic analysis of patients with disorders of cerebral cortical development
Distinct variants affecting differential splicing of TGFBR1 exon 5 cause either Loeys–Dietz syndrome or multiple self-healing squamous epithelioma
Identification of variants in pleiotropic genes causing “isolated” premature ovarian insufficiency: implications for medical practice
Effects of autozygosity and schizophrenia polygenic risk on cognitive and brain developmental trajectories
Using a genetic test result in the care of family members: how does the duty of confidentiality apply?
Clinical Utility Gene Card for: autosomal dominant myotonia congenita (Thomsen Disease)
Germline variants in SMARCB1 and other members of the BAF chromatin-remodeling complex across human disease entities: a meta-analysis
Australians’ views on personal genomic testing: focus group findings from the Genioz study
A pathogenic role for germline PTEN variants which accumulate into the nucleus
Are your covariates under control? How normalization can re-introduce covariate effects
Functional missense and splicing variants in the retinoic acid catabolizing enzyme CYP26C1 in idiopathic short stature
Genome-wide linkage analysis in Spanish melanoma-prone families identifies a new familial melanoma susceptibility locus at 11q
Disease-causing variants in TCF4 are a frequent cause of intellectual disability: lessons from large-scale sequencing approaches in diagnosis
Distress, uncertainty, and positive experiences associated with receiving information on personal genomic risk of melanoma
Tubulinopathies continued: refining the phenotypic spectrum associated with variants in TUBG1
Does ATRX germline variation predispose to osteosarcoma? Three additional cases of osteosarcoma in two ATR-X syndrome patients
Carrier frequency analysis of mutations causing autosomal-recessive-inherited retinal diseases in the Israeli population
RNA analysis of cancer predisposing genes in formalin-fixed paraffin-embedded tissue determines aberrant splicing
Genes flow by the channels of culture: the genetic imprint of matrilocality in Ngazidja, Comoros Islands
Biallelic PADI6 variants linking infertility, miscarriages, and hydatidiform moles
Circulating cell-free nucleic acids: characteristics and applications
Recontacting or not recontacting? A survey of current practices in clinical genetics centres in Europe
Clinical Utility Gene Card For: GALNT3 defective congenital disorder of glycosylation
A decision tree for the genetic diagnosis of deficiency of adenosine deaminase 2 (DADA2): a French reference centres experience
Parent-of-origin effect of hypomorphic pathogenic variants and somatic mosaicism impact on phenotypic expression of retinoblastoma
Psychological outcomes and surgical decisions after genetic testing in women newly diagnosed with breast cancer with and without a family history
Linked homozygous BMPR1B and PDHA2 variants in a consanguineous family with complex digit malformation and male infertility
Re-assessment of multiple testing strategies for more efficient genome-wide association studies
Role of copy number variants in sudden cardiac death and related diseases: genetic analysis and translation into clinical practice
JBS Haldane: an evolutionary geneticist not without controversy
Phenotype of CNTNAP1: a study of patients demonstrating a specific severe congenital hypomyelinating neuropathy with survival beyond infancy
Molecular-genetic characterization of common, noncoding UBASH3A variants associated with type 1 diabetes
Genetic diseases and information to relatives: practical and ethical issues for professionals after introduction of a legal framework in France
RD-Connect, NeurOmics and EURenOmics: collaborative European initiative for rare diseases
Clinical utility gene card: for pseudoxanthoma elasticum
The association between cancer family history and ovarian cancer risk in BRCA1/2 mutation carriers: can it be explained by the mutation position?
New insights from Thailand into the maternal genetic history of Mainland Southeast Asia
Genetic determinants of glycated hemoglobin levels in the Greenlandic Inuit population
γ-glutamyl transpeptidase deficiency caused by a large homozygous intragenic deletion in GGT1
Whole-exome sequence analysis highlights the role of unmasked recessive mutations in copy number variants with incomplete penetrance
Novel variants in Nordic patients referred for genetic testing of telomere-related disorders
Uncovering the genetic lesions underlying the most severe form of Hirschsprung disease by whole-genome sequencing
Genetic predictors of systemic sclerosis-associated interstitial lung disease: a review of recent literature
A novel variant associated with HDL-C levels by modifying DAGLB expression levels: An annotation-based genome-wide association study
Genetics of dementia in a Finnish cohort
A genome-wide characterization of copy number variations in native populations of Peninsular Malaysia
Clinical and genetic spectrum of AMPD2-related pontocerebellar hypoplasia type 9
Clinical Utility Gene Card for: Becker muscular dystrophy
A novel MAP3K7 splice mutation causes cardiospondylocarpofacial syndrome with features of hereditary connective tissue disorder
A randomized controlled study of a consent intervention for participating in an NIH genome sequencing study
ERLIN1 mutations cause teenage-onset slowly progressive ALS in a large Turkish pedigree Turkish
Periodic reanalysis of whole-genome sequencing data enhances the diagnostic advantage over standard clinical genetic testing
Exome sequencing has higher diagnostic yield compared to simulated disease-specific panels in children with suspected monogenic disorders
Homozygous deletion in MYL9 expands the molecular basis of megacystis–microcolon–intestinal hypoperistalsis syndrome
Bi-allelic inactivating variants in the COCH gene cause autosomal recessive prelingual hearing impairment
Reply to Brioude et al
Biallelic inactivating variants in the GTPBP2 gene cause a neurodevelopmental disorder with severe intellectual disability
Incidental inequity
Revisiting Wilms tumour surveillance in Beckwith–Wiedemann syndrome with IC2 methylation loss, reply
Deep phenotyping of speech and language skills in individuals with 16p11.2 deletion
Genetic defects in mtDNA-encoded protein translation cause pediatric, mitochondrial cardiomyopathy with early-onset brain disease
Improved estimation of SNP heritability using Bayesian multiple-phenotype models
Views of rare disease participants in a UK whole-genome sequencing study towards secondary findings: a qualitative study
Understanding the role of the chromosome 15q25.1 in COPD through epigenetics and transcriptomics
How is genetic testing evaluated? A systematic review of the literature
Deep intronic variation in splicing regulatory element of the ERCC8 gene associated with severe but long-term survival Cockayne syndrome
Offering pregnant women different levels of genetic information from prenatal chromosome microarray: a prospective study
Cytochrome c oxidase deficiency, oxidative stress, possible antioxidant therapy and link to nuclear DNA damage
Next-generation sequencing of AV nodal reentrant tachycardia patients identifies broad spectrum of variants in ion channel genes
Fanconi anemia: from DNA repair to metabolism
The RD-Connect Registry & Biobank Finder: a tool for sharing aggregated data and metadata among rare disease researchers
A clinical and molecular characterisation of CRB1-associated maculopathy
Knowledge, experiences and attitudes concerning genetics among retinoblastoma survivors and parents
Psychosocial impact on mothers receiving expanded newborn screening results
Genome-wide association study of Hirschsprung disease detects a novel low-frequency variant at the RET locus
Interpretations of autonomous decision-making in antenatal genetic screening among women in China, Hong Kong and Pakistan
CXorf56, a dendritic neuronal protein, identified as a new candidate gene for X-linked intellectual disability
Patient experience and utility of genetic information: a cross-sectional study among patients tested for cancer susceptibility and thrombophilia
Usefulness of the genetic risk score to identify phenocopies in families with familial hypercholesterolemia?
Clinical utility gene card for McArdle disease
Somatic APC mosaicism and oligogenic inheritance in genetically unsolved colorectal adenomatous polyposis patients
Human beta defensin (HBD) gene copy number affects HBD2 protein levels: impact on cervical bactericidal immunity in pregnancy
Cost of cancer diagnosis using next-generation sequencing targeted gene panels in routine practice: a nationwide French study
Association of metreleptin treatment and dietary intervention with neurological outcomes in Celia’s encephalopathy
Congenital diaphragmatic hernia as a part of Nance–Horan syndrome?
Neuromyopathy with congenital cataracts and glaucoma: a distinct syndrome caused by POLG variants
Identification of 22q13 genes most likely to contribute to Phelan McDermid syndrome
Key apoptotic genes APAF1 and CASP9 implicated in recurrent folate-resistant neural tube defects
Whole-sequence analysis indicates that the Y chromosome C2*-Star Cluster traces back to ordinary Mongols, rather than Genghis Khan
Exome-wide analysis of mutational burden in patients with typical and atypical Rolandic epilepsy
Genetic variant in CACNA1C is associated with PTSD in traumatized police officers
Variants in members of the cadherin–catenin complex, CDH1 and CTNND1, cause blepharocheilodontic syndrome
Genome-wide search for higher order epistasis as modifiers of treatment effects on bone mineral density in childhood cancer survivors
Effect of deliberation on the public’s attitudes toward consent policies for biobank research
Assessing the effectiveness of NICE criteria for stratifying breast cancer risk in a UK cohort
Targeted next generation sequencing in a young population with suspected inherited malignant cardiac arrhythmias
Compound heterozygous SPATA5 variants in four families and functional studies of SPATA5 deficiency
Biallelic variants in KIF14 cause intellectual disability with microcephaly
Human germline gene editing: Recommendations of ESHG and ESHRE European Society of Human Reproduction and Embryology (ESHRE) and the European Society of Human Gene
Responsible innovation in human germline gene editing: Background document to the recommendations of ESHG and ESHRE
Perceptions of legislation relating to the sharing of genomic biobank results with donors—a survey of BBMRI-ERIC biobanks
Older mothers and increased impact of prenatal screening: stable livebirth prevalence of trisomy 21 in the Netherlands for the period 2000–2013
De novo BK channel variant causes epilepsy by affecting voltage gating but not Ca2+ sensitivity
SHOX haploinsufficiency presenting with isolated short long bones in the second and third trimester
Recessive loss of function PIGN alleles, including an intragenic deletion with founder effect in La Réunion Island, in patients with Fryns syndrome
CUGC for Duchenne muscular dystrophy (DMD)
Phenotypic interpretation of complex chromosomal rearrangements informed by nucleotide-level resolution and structural organization of chromatin
Phenotypic characteristics of colorectal cancer in BRCA1/2 mutation carriers
Clinical and experimental evidence suggest a link between KIF7 and C5orf42-related ciliopathies through Sonic Hedgehog signaling
Preconception carrier screening for multiple disorders: evaluation of a screening offer in a Dutch founder population
Functional and association analysis of an Amerindian-derived population-specific p.(Thr280Met) variant in RBPJL, a component of the PTF1 complex Amerindian-derived
Diagnostic challenges in a child with early onset desmoplastic medulloblastoma and homozygous variants in MSH2 and MSH6