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Solid Provides IGNITE DMD Phase I/II Clinical Trial and Pipeline Update

Solid Biosciences has shared an update with the community this morning, including a dosing update in the IGNITE DMD Phase I/II clinical trial, promising long-term biopsy data from prior patients dosed with SGT-001 in the…

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NS Pharma Announces VILTEPSO® Long-Term Trial Data to be Presented at PPMD’s 2021 Virtual Annual Conference

NS Pharma has announced that they will be presenting new, long-term efficacy and safety data from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) at PPMD’s 2021 Virtual Annual Conference next month….

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Pfizer Shares Update on CIFFREO Phase 3 Gene Therapy Trial

Today Pfizer shared an update regarding their Phase 3 gene therapy trial, CIFFREO, with the community. While Pfizer has not yet opened sites within the U.S., the study is underway at 14 trial sites across…

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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, 20201, 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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