Duchenne, Solid and gene therapy

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 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.
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 initial clinical data from INSPIRE DUCHENNE trial
Solid Biosciences (SLDB) announced initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy
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% (N=3) and significant improvements in multiple additional muscle health biomarkers observed support the potential of SGT-003 as a next-generation,
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.
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,
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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 ...
'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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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 ...
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.
Entrada Therapeutics: Cash-Rich Innovator With Asymmetric Upside
Entrada Therapeutics' innovative Endosomal Escape Vehicle platform, poised to revolutionize drug delivery. Click here to read ...
ITF Therapeutics LLC Announces Multiple Data Presentations at 2025 MDA Clinical and Scientific Conference
ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced that seven abstracts have been accepted as poster presentations at the Muscular Dystrophy Association (MDA) Clinical and ...
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 ...
Massillon community rallies to give 7-year-old with muscular dystrophy unforgettable Valentine's Day
Residents came together to deliver thousands of cards, gifts, and thoughtful notes for one very special little boy.