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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March 22, 2019

Take Action before House and Senate Letters Close

Take Action before House and Senate Letters Close If you haven’t done so already please contact your Members of Congress – today – and ask them to support the FY 2020 Duchenne Appropriations Request. The…

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March 20, 2019

Register Today for PPMD’s 2019 Annual Conference!

PPMD’S 2019 ANNUAL CONFERENCE JUNE 26 – JUNE 30, 2019 ORLANDO, FL 2019 marks PPMD’s 25th Annual Conference. No other Duchenne conference comes close to the experience of the PPMD Annual Conference. Families, physicians, researchers, caregivers,…

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March 19, 2019

Cure SMA & PPMD Announce Strategic Collaboration with Prometheus Research

PPMD is thrilled to announce our strategic collaboration with Cure SMA, one of the world’s leading rare disease patient advocacy organizations. Like PPMD, Cure SMA is looking to advance data collection and analysis on their…

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March 13, 2019

Updates from PPMD’s 2019 Gene Therapy Policy Forum

During PPMD’s Annual Advocacy Conference we had the opportunity to host a Gene Therapy Forum with expert clinicians, researchers, industry stakeholders, and government representatives taking part in the discussion. This meeting continues PPMD’s in-depth look…

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March 11, 2019

Respiratory Management in Duchenne

Improved respiratory (breathing) management over the past 10 years has significantly improved the quality and length of life for people living with Duchenne. In October 2018, articles discussing specific areas of Duchenne care were released. One…

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March 5, 2019

Action Alert! Continue Progress in the Fight Against Duchenne

Thanks to the work of our Duchenne community over the past two decades, Congress has regularly supported important programs and policies to advance research, public health, drug development, and other Duchenne priorities. To ensure that we…

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