As an early funder of Avidity Biosciences, we are pleased to share that Novartis has announced that the FDA has accepted the Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) for Priority […]
Proposed text: CureDuchenne was an early funder of Sarepta Therapeutics, and we are pleased to share that the US FDA has accepted supplemental New Drug Applications (sNDAs) for AMONDYS 45 […]
As a founding partner and initial funder of the Collaborative Trajectory Analysis Project (cTAP), CureDuchenne is proud to support an important advancement for the Duchenne community: a novel prognostic score that helps […]
Emerging guidance offers cautious optimism—here’s what parents and caregivers should know For years, families affected by Duchenne muscular dystrophy have heard a consistent message: “be cautious with exercise, don’t overdo it.” That guidance came from a […]
What Sarepta Therapeutics’ sNDA submission means for Duchenne Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 […]
ITF shared a community letter and presented new data, including long-term safety observations based on data from the company’s ongoing open-label extension study in patients with Duchenne muscular dystrophy treated with […]
REGENXBIO GENE THERAPY RGX-202, an investigational gene therapy for Duchenne, continues to show a favorable safety profile with no serious adverse events, no liver injury signals, and reductions in key […]
Santhera Pharmaceuticals announced results from the ongoing Guardian study in Duchenne, and showed that AGAMREE had comparable effectiveness compared to historical cohorts taking prednisone or deflazacort, based on time to […]
As an early funder of Entrada Therapeutics, CureDuchenne is pleased to share that after the independent Data Monitoring Committee reviewed the safety data from Cohort 1 of the ELEVATE-44-201 trial […]
