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...
Solid Biosciences Provides Update on INSPIRE DUCHENNE Clinical Trial Progress and Planned Regulatory Discussions
Solid Biosciences announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial. The Company also provided an update to the planned...
Five-Year Data of AGAMREE (vamorolone) in Patients with DMD Show Improved Safety Profile with Comparable Effectiveness to Standard of Care Corticosteroids
Santhera reported positive topline results from an analysis of long-term data, including first assessments from the ongoing, open-label, multicenter GUARDIAN study evaluating AGAMREE (vamorolone)...