September 27, 2021

Sarepta Therapeutics Plans to Initiate Part B of the MOMENTUM Study of SRP-5051 in Patients with Duchenne Following Positive Interactions with FDA

PPMD is pleased to share that Sarepta Therapeutics plans to initiate Part B of the MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy following positive interactions with the U.S. Food and Drug Administration…

Learn More

September 24, 2021

Capricor Therapeutics Announces Positive Final Data From its Phase II HOPE-2 Trial in Patients with Duchenne Muscular Dystrophy Treated with CAP-1002

PPMD is pleased to share that Capricor Therapeutics announced today positive final data from the HOPE-2 randomized, double-blind, placebo controlled clinical trial using CAP-1002 to treat patients with Duchenne. The HOPE-2 clinical trial met its…

Learn More

August 17, 2021

Solid provides updates on IGNITE DMD and SGT-003, next generation gene therapy pipeline initiative

Solid Biosciences has shared an update with the community on dosing as part of the IGNITE DMD Phase I/II clinical trial, developments for next generation gene therapy pipeline initiative SGT-003 and their collaboration with Ultragenyx….

Learn More

August 16, 2021

Edgewise Receives Fast Track Designation for EDG-5506 in Patients with Becker muscular dystrophy

Edgewise Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for…

Learn More

August 12, 2021

NS Pharma Shares Community Letter

August 12, 2020, marked the approval of Viltepso, the 4th FDA approved therapy for Duchenne. NS Pharma has posted the following community letter marking the occasion. Today we recognize the one-year anniversary of this approval….

Learn More

July 6, 2021

Recently Reported Updates from PPMD’s 2021 Virtual Annual Conference

PPMD’s 2021 Virtual Annual Conference: Recently Reported Updates As a new addition to PPMD’s 2021 Virtual Conference, PPMD invited industry partners to share new data with the community live during the Conference in a series…

Learn More

June 26, 2021

Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker

This afternoon at PPMD’s Virtual Annual Conference, Italfarmaco shared Phase II Study results from the Givinostat trial in patients with Becker. They also issued a press release below. Although they did not meet the primary…

Learn More

June 11, 2021

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording)

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar update on June 2, 2021. Sarepta was invited to present recent updates from their ongoing clinical…

Learn More

June 1, 2021

Santhera & ReveraGen Announce Positive & Statistically Significant Topline Results in VISION-DMD Study

Santhera and ReveraGen today announced positive and statistically significant results from the 24-week readout of the VISION-DMD study of vamorolone in Duchenne. The companies report that study demonstrated strong efficacy across primary and secondary endpoints…

Learn More

May 21, 2021

WATCH: ASP0367 Development Program Update (Webinar Recording)

Astellas Pharma joined PPMD for a webinar on May 19, 2021 to provide an update on the company’s ASP0367 development program. If you missed the live event, the recording can be found below.    …

Learn More