December 23, 2024

Life is about time and timing when living with Duchenne

As a family living with Duchenne, time is everything to us. Decades of trials and research have paved the way for a new era, ushering in a reality with a growing landscape of both approved…

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December 19, 2024

WATCH: Sevasemten – CANYON Trial Topline Results (Webinar Recording)

Edgewise Therapeutics recently joined PPMD for a breaking news community webinar, during which Edgewise provided an update on the recently released topline results from its CANYON double-blind, randomized, placebo-controlled design to investigate the effect of…

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December 19, 2024

Support PPMD’s commitment to moving the science further, faster

Greetings from Indiana! I write today letting you know that thanks to a generous pledge from a local family I know well, a $100,000 matching gift will be made to PPMD. That means every dollar…

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December 18, 2024

Percheron Announces Termination of Phase 2B Study of Avicursen for Duchenne

PPMD is disappointed to learn of Percheron Therapeutics’ recent decision to terminate the company’s ongoing Phase 2b randomized, placebo-controlled trial of avicursen (ATL1102) in non-ambulatory individuals living with Duchenne following the release of topline six-month…

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December 17, 2024

Your gift to PPMD is a commitment to accelerating progress

As a pediatric neurologist who takes care of children with neuromuscular diseases, I’ve had the privilege of witnessing first-hand the benefit many of our therapies can have for individuals living with Duchenne. Your belief in…

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December 17, 2024

PPMD Convenes GI, GU, and Swallowing Experts for Two-Day Workshop

Earlier this month, PPMD convened experts from across the country in Columbus, Ohio for a two-day workshop focused on the development of care standards for gastrointestinal, urological, and swallowing issues in Duchenne and Becker.  As…

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December 16, 2024

PepGen Announces Clinical Hold in the U.S. on IND Application to Initiate CONNECT2-EDO51 Phase 2 Study of PGN-EDO51

PPMD is disappointed to learn that PepGen Inc. has received a full clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application to initiate the CONNECT2-EDO51 clinical…

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December 16, 2024

Edgewise Therapeutics Announces Positive Topline Results from CANYON Phase 2 Trial of Sevasemten in Individuals with Becker

Edgewise Therapeutics, Inc., has announced positive topline results from the double-blind, randomized, placebo-controlled Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to…

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December 11, 2024

Advance the Research Evolution: Introducing PPMD’s Dystrophinopathy Clinical Research Network

In 1984, the word “Duchenne” entered my world. The progress we have made since then is astounding. For decades, we have united in our unwavering commitment to end Duchenne, and with recent achievements in this…

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December 6, 2024

Catch up on PPMD Together: Virtual Edition – A Gathering of Families for Connection, Support, and Growth

Last month, families from around the world came together for PPMD Together: Virtual Edition. This unique virtual gathering provided families a space to connect, share their experiences, and find support from one another as they…

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