Avidity Biosciences Announces U.S. Managed Access Program (MAP) for Investigational Therapy del-zota (Exon 44 Skipping)
Avidity Biosciences announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to...
Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature
Satellos announced the publication in Nature Communications of new research from a scientific team at the Ottawa Hospital Research Institute (OHRI) that validates the...
Solid Biosciences Awarded Innovation Passport Designation Under the UK Innovative Licensing and Access Pathway for SGT-003, an Investigational Gene Therapy for Duchenne Muscular Dystrophy
Solid Biosciences announced that SGT-003, the Company’s investigational gene therapy for Duchenne muscular dystrophy (Duchenne), has been granted an Innovation Passport under the new...
Sarepta’s DMD Exon-Skipping Therapies Fail Confirmatory Study, Stock Price Down
Sarepta Therapeutics' difficulties only get worse. In patients with Duchenne muscular dystrophy, the business found that their exon-skipping treatments, Vyondys 53 and Amondys 45,...
PPMD Provides $400,000 in Funding to MyoGene Bio for MyoDys Gene Editing Platform
PPMD has provided $400,000 in funding to MyoGene through PPMD Venture Pathways, the organization's venture-philanthropy initiative that provides industry funding to accelerate therapeutic development...