Muscular dystrophies represent a group of disorders characterized by progressive muscle degeneration and weakness. An important subgroup are the dystrophin-related muscular dystrophies caused by ...

lumc.nl Objective Becker muscular dystrophy (BMD) is characterised by broad clinical variability. Ongoing studies exploring dystrophin restoration in Duchenne muscular dystrophy ask for better ...

Dyne Therapeutics on Tuesday unveiled new data from its Phase I/II DELIVER trial, demonstrating that its investigational oligomer therapy DYNE-251 can induce record levels of dystrophin expression in ...

Sarepta's Elevidys approval, revenue growth projections, and competitive position make it a strong investment in DMD ...

While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, ...

A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in ...

Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, the ...

2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with ...

Satellos Bioscience Inc. ("Satellos" or the "Company") (TSX: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle ...

The boy who inspired "Michael's Cause" at age 4 is now a young adult whose family continues to fight for research of Duchenne Muscular Dystrophy.

The U.S. Food and Drug Administration granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping DMD ...