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…

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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….

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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…

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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…

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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…

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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…

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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.    …

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May 14, 2021

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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May 12, 2021

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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May 4, 2021

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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