Solid Biosciences (SLDB) is hoping to advance a next-generation gene therapy for the treatment of patients with Duchenne Muscular Dystrophy [DMD]. This would be with a gene therapy candidate by the ...

There is an ongoing phase 3 study, known as HOPE-3, deploying the use of CAP-1002 for the treatment of patients with Duchenne Muscular Dystrophy; Interim analysis expected Q4 of 2023. A Type-B FDA ...

Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for ...

TORONTO--(BUSINESS WIRE)--Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the ...

MEMPHIS, Tenn.--(BUSINESS WIRE)--GTx, Inc. (Nasdaq: GTXI) today announced the publication in the journal Human Molecular Genetics of results from preclinical studies supporting the potential efficacy ...

DMD is predominately caused by out-of-frame deletions in the dystrophin gene, which results in absent or defective dystrophin protein, leading to progressive and irreversible muscle function loss. The ...

- Analysis showed improvement in muscle morphology and increased levels of regeneration in muscle groups, including the diaphragm, quadriceps, and calf - No adverse events and no significant changes ...

Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children's Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in ...

Ronald Berenson, serial entrepreneur, is heading up DMD Therapeutics. (CoMotion Photo / Conrado Tapado) DMD Therapeutics, a biotech startup that was spun out of the University of Washington by biotech ...

Researchers have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a ...

Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of ...