Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), an exon-skipping experimental therapeutic for […]
Newport Beach, Calif. (May 7, 2024) – CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, announced its FUTURES National Conference will be held from May 23-26 in Orlando, Florida. FUTURES is a four-day event focused on bringing education, connection and hope to the Duchenne and Becker community. The annual event will take place as a hybrid event, offering attendees a chance to participate both online and in-person, at the JW Marriott Orlando, Grande Lakes.
Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
Fat embolism syndrome (FES) is a potentially life-threatening condition that can be triggered by bone fractures.
REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51. In part B of the Phase 2 MOMENTURM study, ambulatory and […]
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for DMD in the UK.
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
