'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 ...
Health.com2d
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 ...
I'm dying of a fatal disease. Bureaucrats shouldn’t decide how doctors can try to save me
I'm fighting a fatal disease that took the life of my brother. I work in biotech and know there are medical advances that ...
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 ...
Labroots4d
New Insights Into Duchenne Muscular Dystrophy Reveal Therapeutic Options
Muscular dystrophy is a disease caused by a genetic mutation. The muscles of muscular dystrophy patients weaken.. | Genetics ...
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 ...
Business Wire4d
Satellos Announces Upcoming Presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a public biotech company developing new ...
STAT4d
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 ...
Solid Biosciences Stock Surges On Upbeat Duchenne Muscular Dystrophy Gene Therapy Data
Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.