December 20, 2023

CDCC Spotlight: Riley Hospital for Children

The Little Things There are many ways the team at Riley Children’s Certified Duchenne Care Center go the extra mile. One such way is by providing patients with individualized toys and activities for each of…

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December 19, 2023

Support Every Future: Your Gift to PPMD Doubles Until December 31st

Many of you know as well as we do that receiving a diagnosis of Duchenne is both crushing and overwhelming. We received the diagnosis for both of our sons a little over two years ago…

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December 15, 2023

Wave Life Sciences Announces Initiation of Dosing in Phase 2 Duchenne Clinical Trial of WVE-N531

Wave Life Sciences recently shared that the company has initiated dosing and has fully enrolled the Phase 2 FORWARD-53 trial of investigational WVE-N531, a potential exon skipping therapy targeting people with Duchenne amenable to exon…

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December 14, 2023

Your Gift MATCHED Through 12/31 When You Support PPMD’s Fight for Every Future

In 1992, I was diagnosed with Duchenne at two years old, significantly earlier than the typical diagnosis age of 6 or 7 at that time, due to a family history. In the 90’s, our understanding…

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December 12, 2023

Watch: Community Update on the New INSPIRE DUCHENNE Clinical Study of SGT-003 (Webinar Recording)

Solid Biosciences recently joined PPMD for a community webinar to provide an overview of INSPIRE DUCHENNE, a first-in-human clinical study evaluating SGT-003, a next-generation microdystrophin gene therapy candidate for the treatment of Duchenne. The Solid…

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December 11, 2023

Register Now for PPMD’s 2024 Advocacy Conference

Join us in Washington, DC, March 3–5, 2024, as we continue to fight for every future. Each year, PPMD ensures that the Duchenne community is at the forefront of the hearts and minds of Congressional…

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December 7, 2023

Your Gift Today Funds PPMD’s Fight for Every Future

For over three decades, my life’s work has centered around scientific research aimed at making a positive impact on the lives of individuals living with Duchenne. I have witnessed breakthroughs as therapeutic options moved from…

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December 5, 2023

PTC Therapeutics Shares Update on Translarna™ Regulatory Activities

PTC Therapeutics, Inc. has shared an update on regulatory activities in Europe and the United States for Translarna™ (ataluren), a treatment for Duchenne patients with nonsense variants.  Europe: European Medicines Agency (EMA) Update Following the…

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December 4, 2023

Winter Safety, Prevention & Preparedness

With winter quickly approaching, PPMD wants to remind you about the importance of safety, prevention, and being prepared for potential emergencies such as falls and fractures, acute respiratory illness, and other unpredictable situations.  SAFETY: Snow…

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December 1, 2023

Watch: AGAMREE® Approval with Catalyst Pharmaceuticals, Inc. (Webinar Recording)

PPMD was recently joined by Catalyst Pharmaceuticals to review the recent FDA approval of AGAMREE (vamorolone), a new steroid approved for the treatment of Duchenne.  Amy Grover, Catalyst’s Senior Director of Patient Advocacy, introduced the…

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