Blog

WATCH: Viltepso – A Treatment Option for Patients Amenable to Exon 53 Skipping (Webinar Recording)

NS Pharma joined PPMD for a webinar on April 22, 2021 to provide an update about VILTEPSO™, a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to…

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Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051

Sarepta has announced positive results from Part A of the MOMENTUM study (Study 5051-201), a Phase 2 clinical trial of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. Part…

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Santhera and ReveraGen Announce New 2.5-year Treatment Data with Vamorolone in Duchenne

Today Santhera and ReveraGen shared 2.5-year treatment data with vamorolone in Duchenne. In the announcement below the companies reported on data from 46 patients who have participated in two open-label extension studies covering 2.5 years,…

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Webinar Q&A: Gene Therapy – Turning Obstacles into Possibilities

Last week, PPMD held a webinar to discuss the lessons learned in Gene Therapy, current challenges for our community, and what the future holds for these promising treatments. After opening with an insightful presentation from…

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WATCH: Gene Therapy – Turning Obstacles into Possibilities (Webinar Recording)

On April 20, 2021, PPMD’s Pat Furlong & Eric Camino were joined by Drs. Nagaraju and Shieh for an interactive panel discussion on the lessons learned in Gene Therapy, current challenges for our community, and…

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WATCH: Transforming the Treatment Paradigm with Dyne Therapeutics (Webinar Recording)

Dyne Therapeutics joined Parent Project Muscular Dystrophy (PPMD) for a webinar on March 31, 2021 to provide an overview of the company’s mission, its novel FORCE platform designed to revolutionize drug delivery to muscle, and…

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Register Today for PPMD’s 2021 Annual Conference — Virtual Edition!

PPMD’s Annual Conference – Virtual Edition Registration is now open for PPMD’s 2021 Virtual Annual Conference! 2021 remains an extraordinary time for our global community. We are getting closer to safer days ahead and we want…

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WATCH: Santhera Pharmaceuticals Duchenne Drug Development Update (Webinar Recording)

Santhera Pharmaceuticals joined Parent Project Muscular Dystrophy (PPMD) for a webinar on March 24, 2021 to provide an update to the community about the investigational therapy vamorolone, the use of steroids to treat Duchenne muscular…

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WATCH: Solid Biosciences Presents Safety & Efficacy Update (Webinar Recording)

On March 16, 2021, Solid Biosciences joined PPMD for a webinar to provide a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with…

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PPMD’s Certified Duchenne Care Center Program – 2020 Impact & Progress

PPMD’s Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the CDC Care Considerations, available and accessible to as many families as possible, and to strengthen clinical trial…

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