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 ...

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

Satellos Bioscience Inc. ("Satellos" or the "Company") , a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, ...

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

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

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

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.

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, ...

Avidity supports World Duchenne Awareness Day, International Myotonic Dystrophy Awareness Day, FSHD Society Walk & Roll to Cure FSHD and Global Genes Week in RARE SAN DIEGO, Sept. 5, 2024 /PRNewswire/ ...

Somite Therapeutics, a tech-bio company harnessing big data and AI to pioneer novel cell replacement therapies, and OmniaBio Inc., a technology-driven cell and gene therapy CDMO founded by CCRM, today ...