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Introduction/Aims
Facioscapulohumeral muscular dystrophy (FSHD) is a slowly progressive muscular dystrophy without approved therapies. In this study we evaluated whether locally acting ACE‐083 could safely increase muscle volume and improve functional outcomes in adults with FSHD.
Methods
Participants were at least 18 years old and had FSHD1/FSHD2. Part 1 was open label, ascending dose, assessing...
This case report describes a young boy with concomitant genetically-confirmed Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy with a novel dystrophin mutation in exon 6 and a D4Z4 fragment of 31kb. This child presented with a more severe phenotype than expected for either individual disease process and underscores the role for thorough diagnostic investigation in identifying...
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