Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy.
Read the full news release HERE Capricor Therapeutics, which received early funding from CureDuchenne, has announced positive 3-year results from the HOPE-2 open-label extension study. Individuals receiving CAP-1002 continue to […]
As a funder of hC Bioscience, we’re happy to have them announcing their tRNA-based approach for targeting nonsense mutations at our annual Futures conference.
As early investors in Dyne Therapeutics, we are pleased to share they reported positive updates in their next-generation exon skipping program for Duchenne amenable to skipping exon 51. In their […]
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.
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)
Meet members of the ITF Therapeutics team to learn more about their organization and next steps to support the availability of DUVYZAT.
