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SMCHD1 has separable roles in chromatin architecture and gene silencing that could be targeted in disease
T cell responses to dystrophin in a natural history study of Duchenne muscular dystrophy
Occurrence of symptoms in different stages of Duchenne muscular dystrophy and their impact on social participation
Compliance to DMD care considerations in the Netherlands
Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy
Dnmt3bregulates DUX4 expression in a tissue-dependent manner in transgenic D4Z4 mice
Decision-making and selection bias in four observational studies on Duchenne and Becker muscular dystrophy
Decision-making and selection bias in four observational studies on Duchenne and Becker muscular dystrophy
Downregulation of miRNA-29,-23 and-21 in urine of Duchenne muscular dystrophy patients
Smchd1 haploinsufficiency exacerbates the phenotype of a transgenic FSHD1 mouse model
Deep characterization of a common D4Z4 variant identifies biallelic DUX4 expression as a modifier for disease penetrance in FSHD2
DUX4 induces a transcriptome more characteristic of a less-differentiated cell state and inhibits myogenesis
PABPN1-Dependent mRNA Processing Induces Muscle Wasting
PABPN1-Dependent mRNA Processing Induces Muscle Wasting
Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9
Intrinsic Epigenetic Regulation of the D4Z4 Macrosatellite Repeat in a Transgenic Mouse Model for FSHD
Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2
Generation of Isogenic D4Z4 Contracted and Noncontracted Immortal Muscle Cell Clones from a Mosaic Patient A Cellular Model for FSHD
Modulation of estrogen signaling in hepatic and vascular tissue