Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
Sarepta Therapeutics announced topline results from EMBARK
The U.S. FDA announced today its decision to approve AGAMREE (vamorolone), a new steroid for individuals with Duchenne muscular dystrophy 2 years or older. This approval comes largely based on […]
The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for AGAMREE’s approval in Europe.
– GRAND CANYON is the first pivotal study of an investigational therapy for Becker–– CANYON, the initial Phase 2 cohorts, is fully enrolled – BOULDER, Colo.–(BUSINESS WIRE)– Edgewise Therapeutics, Inc. (Nasdaq: […]
See Previous News Release – Initiation of Phase 1 clinical trial marks Entrada’s transition into a clinical company – – Data anticipated in the second half of 2024 – BOSTON, […]
FibroGen announced topline data from their Phase 3 trials of their novel antifibrotic agent, pamrevulmab, a connective tissue growth factor antibody for the treatment for ambulatory individuals with Duchenne. While pamrevlumab was generally safe and well tolerated, the study unfortunately did not meet the primary endpoint. Pamrevlumab also failed to meet its primary endpoint in non-ambulatory individuals earlier this year.
– First participant is expected to be dosed in September 2023 with data anticipated in the second half of 2024 – – Cash runway extended through the end of 2025 […]
While there is currently no cure for Duchenne muscular dystrophy, gene therapy has the potential to target the underlying genetic cause of many mono genetic diseases and could potentially benefit […]
