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Antisense oligonucleotide-mediated disruption of HTT caspase-6 cleavage site ameliorates the phenotype of YAC128 Huntington disease mice
Longitudinal Assessment of creatine kinase, creatine/creatinine(ratio), and myostatin as monitoring biomarkers in Becker muscular dystrophy
Longitudinal assessment of creatine kinase, creatine/creatinineratio, and myostatin as monitoring biomarkers in Becker muscular dystrophy
Longitudinal assessment of creatine kinase, creatine/creatinine ratio, and myostatin as monitoring biomarkers in Becker muscular dystrophy
Longitudinal assessment of creatine kinase, creatine/creatinine ratio and myostatin as monitoring biomarkers in Becker muscular dystrophy
Huntington disease gene expression signatures in blood compared to brain of YAC128 mice as candidates for monitoring of pathology
Broad phenotype of cysteine-altering NOTCH3 variants in UK Biobank CADASIL to nonpenetrance
Naturally occurring NOTCH3 exon skipping attenuates NOTCH3 protein aggregation and disease severity in CADASIL patients
Implications of increased S100 beta and Tau5 proteins in dystrophic nerves of two mdx mouse models for Duchenne muscular dystrophy
Progression and Classification of Granular Osmiophilic Material (GOM) Deposits in Functionally Characterized Human NOTCH3 Transgenic Mice
Natural disease history of the D2-mdx mouse model for Duchenne muscular dystrophy
Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model
Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model
Natural disease history of the dy(2J) mouse model of laminin alpha 2 (merosin)-deficient congenital muscular dystrophy
Detailed natural history of the mdx-DBA model
Natural disease history of mouse models for limb girdle muscular dystrophy types 2D and 2F
New function of the myostatin/activin type I receptor (ALK4) as a mediator of muscle atrophy and muscle regeneration
New function of the myostatin/activin type I receptor (ALK4) as a mediator of muscle atrophy and muscle regeneration