April 15, 2019

Race Athlete Spotlight: Enzo Figueres

Enzo Figueres is an avid mountain biker but will be participating in his first road bike tour this May – with his son in a bike trailer! – as part of the Race to End…

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April 15, 2019

FibroGen Receives U.S. FDA Orphan Drug Designation for Pamrevlumab for the Treatment of Duchenne

FibroGen announced that FDA has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of people with Duchenne. According to their press release, all 21 non-ambulatory Duchenne patients in their ongoing…

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April 11, 2019

79 Members of Congress Send Letter of Strong Support for Duchenne Priorities for Next Year’s Budget

As a result of 143 meetings with Members of Congress in Washington DC, and over 2,000 action alert emails sent by this community, we are pleased to report a very successful outcome for the Duchenne…

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April 10, 2019

Unionville Run For Our Sons 5K Celebrates 10-Year Anniversary

Saturday, April 6 marked the 10th Annual Unionville Run For Our Sons 5K – a local DIY Race event hosted by the Johnson family benefiting Parent Project Muscular Dystrophy (PPMD) and our fight to end…

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April 8, 2019

Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline for Duchenne

Audentes Therapeutics announced that it is expanding its scientific platform and beginning development of new AAV-based genetic medicines for Duchenne. Audentes is a leading AAV-based genetic medicines company based in San Francisco. The company is…

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April 5, 2019

Catabasis Shares March 2019 Newsletters

Catabasis shared the latest edition of their community newsletter (also available in Spanish), including an update on their currently recruiting Phase 3 PolarisDMD trial for edasalonexent in Duchenne. Download the Catabasis newsletters: English Spanish

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April 4, 2019

End Duchenne Tour Recap: Omaha, NE

PPMD had the opportunity to travel to Omaha, NE on March 30 for our second End Duchenne Tour stop of 2019. We were joined by over 50 people including 18 families, 9 industry partners, and 10…

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April 3, 2019

WATCH: Italfarmaco Provides Givinostat Update – Webinar Recording

On Wednesday, March 27, 2019, Italfarmaco joined Parent Project Muscular Dystrophy for a webinar to update the community about Italfarmaco’s development of Givinostat and currently enrolling Phase 3 confirmatory study in Duchenne muscular dystrophy. If…

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March 28, 2019

Sarepta Announces Positive Expression Results from the Casimersen (SRP-4045) Arm of ESSENCE Study

Sarepta Therapeutics announced positive results from its interim analysis of muscle biopsies at week 48, comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. ESSENCE is a global, randomized double-blind,…

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March 25, 2019

Sarepta Provides Micro-dystrophin Gene Therapy Update

Sarepta Therapeutics hosted a webcast earlier this morning, providing an update on encouraging 9-month functional and creatine kinase (CK) data from baseline for the original four patients in the Phase 1 open-label study of Sarepta’s…

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