December 30, 2023

Give Before Midnight Tomorrow. Fight for Every Future!

The days leading up to the New Year are always a time of reflection for me. Since 1994 when my mother founded PPMD, my life has been intertwined with the organization. As we head into…

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December 27, 2023

Your Gift to Support PPMD’s Gene Therapy Initiative Goes Twice as Far Now Through 12/31

My son Connor’s diagnosis of Duchenne turned my family’s life upside down. In order to prioritize Connor’s comfort and quality of life we moved from Chicago to Florida. We’ve changed jobs, schools, and care teams,…

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December 22, 2023

Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label

Sarepta Therapeutics, Inc. today announced the submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS to expand its labeled indication. The company also shared that it completed the Phase 3 EMBARK…

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December 21, 2023

PPMD & Community Partners Urge Timely, Transparent FDA Review of EMBARK Data

Today, PPMD joined the Best Day Ever Foundation, CureDuchenne, the Jett Foundation, the Little Hercules Foundation, The Muscular Dystrophy Family Foundation, and Team Joseph in sending a letter to the Federal Drug Administration (FDA) regarding…

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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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