DMD treatment SMT-01, a cell replacement therapy, received FDA orphan drug and rare pediatric disease designations.

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

AI-driven biotech Somite.ai scores dual FDA designations for its novel treatment, potentially fast-tracking hope for families ...

Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...

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

that contains selected domains of the dystrophin protein present in normal muscle cells. The FDA granted traditional approval for ambulatory individuals 4 years of age and older with DMD with a ...

Having already scooped up the U.S. rights to Capricor Therapeutics’ late-stage Duchenne muscular dystrophy (DMD) therapy, ...

The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the ...

The cells are not thought to engraft into muscle, but exert their effects by ... which sees CAP-1002 as an add-on therapy to current treatments for DMD, including steroids and exon-skipping ...

DMD is a progressive muscle disease due to muscle degeneration leading to severe physical disability and shortened lifespan. In Uganda, it is estimated to affect 230 boys every year. DMD is caused ...

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

SUZHOU, China I 9, 2024 I On 06, 2024, GenAssist Ltd (GenAssist), announced the first DMD patient dosed with its base editing drug, ...