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What To Know About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is ...
'It's cruel that I cannot get muscular dystrophy drug'
A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available ...
STAT5d
Duchenne gene therapy from Solid Biosciences produces promising results in early study
Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...
Solid says early data suggest ‘differentiated’ Duchenne gene therapy
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...
Genetic mechanisms underlying Duchenne muscular dystrophy point to potential treatments
Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...
Minneapolis parents share journey of having two sons with rare muscle-wasting disorder
Caleb Kasner and his brother Duncan Kasner move around a little differently than average kids. The two brothers have Duchenne ...
Massachusetts families, lawmakers fighting to keep NIH funding for life-saving medical research
Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.
The Pharma Letter4d
Solid Biosciences surges on Duchenne therapy success
Boston, USA-based biotech Solid Biosciences saw its shares rocket 36% to $5.48 yesterday, after the precision genetic ...
Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003
Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin expression of 110% ...
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What To Know About Duchenne Muscular Dystrophy (DMD)
Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...