Duchenne, Solid and gene therapy

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 Sarepta Therapeutics’ Elevidys.
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 early sign of promise
Solid Bio surges after trial data for lead asset in Duchenne
Shares of Solid Biosciences (NASDAQ:SLDB) jumped ~88% in the premarket on Tuesday after the gene therapy developer reported promising early results from a Phase 1/2 trial for its lead asset SGT-003 in Duchenne muscular dystrophy,
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 medicines for neuromuscular and cardiac diseases company announced positive initial data from the Phase I/II INSPIRE DUCHENNE trial evaluating SGT-003.
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%
Solid Biosciences Shares Rise After Positive Trial Data for SGT-003 in Duchenne MD
Solid Biosciences shares were up 50% to $6.06 after the company said it saw positive initial data from a Phase 1/2 trial evaluating SGT-003, a gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy.
'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 ...
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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 ...
FiercePharma18h
Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...
Labroots3d
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 ...
Entrada Therapeutics: Cash-Rich Innovator With Asymmetric Upside
Entrada Therapeutics' innovative Endosomal Escape Vehicle platform, poised to revolutionize drug delivery. Click here to read ...
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.
Business Wire3d
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 ...
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 ...