gene therapy, Solid and Duchenne

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 Biosciences’ stock soars on early success in DMD gene therapy trial
The trial was able to elicit more than double the microdystrophin gene in patients after 90 days of treatment.
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 Bio's stock soars as early DMD gene therapy data beat expectations
Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has surged back into the Duchenne muscular dystrophy gene therapy race. The biotech reported expectation-busting phase 1 data on its next-generation candidate,
Solid Biosciences Reports Positive Data from DMD Gene Therapy Trial
Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively to muscle cells.
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 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.
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 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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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. | ...
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Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
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 ...
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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 ...