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Catabasis Connection – October 2020
This month’s Catabasis Connection:
SIDEROS trial interim analysis update from Santhera
Dear U.S. Duchenne community, As you know from our previous updates, the SIDEROS clinical trial is fully enrolled and Santhera has been working with the Food and Drug Administration (FDA) […]
Letter to the Duchenne Community: FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial
Dear Duchenne Community, We are pleased to announce that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on IGNITE DMD, our Phase I/II clinical trial. We […]
PFIZER RECEIVES FDA FAST TRACK DESIGNATION FOR DUCHENNE MUSCULAR DYSTROPHY INVESTIGATIONAL GENE THERAPY
October 01, 2020 NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track […]
Sarepta Therapeutics Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001, its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
–Results demonstrate continued safety and tolerability of SRP-9001 in four participants with Duchenne — –All four participants demonstrated improvements in NSAA scores compared to baseline and showed a durable response two years after administration of SRP-9001 — CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) — Sarepta […]
Updates from World Muscle Society – 2020
CureDuchenne Congratulates Dyne Therapeutics on Its Initial Public Offering
Nonprofit Provided Early-Stage Funding to Accelerate Treatments for Duchenne Muscular Dystrophy Newport Beach, CA (September 25, 2020) – CureDuchenne, a leading global nonprofit dedicated to finding and funding a cure […]
CureDuchenne Webinar: Italfarmaco Givinostat Program Update
Dr. Paolo Bettica’s update about the Italfarmaco Givinostat development program includes progress on the Italfarmaco studies about Givinostat in Duchenne and Becker Muscular Dystrophy then takes your questions about the […]
Santhera Announces Publication of Long-Term Clinical Data with Vamorolone in Patients with Duchenne Muscular Dystrophy
Pratteln, Switzerland, September 22, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces that partner ReveraGen Biopharma Inc. and their academic collaborators have published new open-label, long-term clinical data on the safety, tolerability […]