REGENXBIO provided a clinical and regulatory update on their Duchenne muscular dystrophy program highlighting progress in dose selection for their upcoming pivotal study which is expected to begin dosing patients […]
The Duchenne muscular dystrophy community recently faced a profound loss—a young participant in Pfizer’s Phase 2 DAYLIGHT study (for boys 2 years to less than 4 years of age) passed away due to cardiac arrest, over a year after receiving an investigational micro-dystrophin gene therapy aimed at treating Duchenne.
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.
Capricor Therapeutics Announces Positive Type-B Meeting with FDA for CAP-1002 Program for Duchenne Muscular Dystrophy
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)
FDA Approves Duvyzat (givinostat) for Duchenne Muscular Dystrophy Treatment.
PTC Therapeutics Provides Key Regulatory Updates
Here’s What You Missed at the MDA Clinical and Scientific Conference 2024
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
