January 9, 2024

Join us at PPMD’s 30th year meetings and events

Happy New Year! As PPMD celebrates its 30th anniversary in 2024, we’re thrilled to announce the expansion of our events and meetings dedicated to accelerating research, enhancing care and quality of life, and fostering connections…

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January 8, 2024

PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51

PepGen has announced that the first participant has been dosed in its Phase 2 CONNECT1-EDO51 study, which will evaluate the safety and tolerability of PGN-EDO51, the company’s next-generation conjugated-PMO exon skipping therapy for the treatment…

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January 6, 2024

Race to End Duchenne Celebrates Runners During Walt Disney World Marathon Weekend

The Race to End Duchenne team is in The Most Magical Place on Earth this weekend to participate in Walt Disney World® Marathon Weekend, presented by State Farm. Make Every Day Count  Michael (Mike) Napoli,…

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January 5, 2024

The Importance of Assisted Cough

It’s winter, which means respiratory virus season is upon us. Those living with Duchenne and Becker may find themselves needing more support to help clear thick secretions that often accompany respiratory illness. Using assisted cough…

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January 3, 2024

Thank You for Fighting for Every Future With Us

As we enter the New Year, my heart is filled with profound gratitude as I am once again reminded of the immense generosity of our Duchenne community. For the past three decades, you have supported…

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January 3, 2024

Dyne Therapeutics Announces Positive Initial Clinical Data from DELIVER Trial in Duchenne Patients

Dyne Therapeutics today announced positive initial clinical data from its DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. Dyne-251 is an exon skipping product that combines a PMO…

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December 31, 2023

Give by midnight to double your impact in the fight for every future

As we prepare for the ball to drop and the confetti to fall, I am struck by the significance that this will be PPMD’s 30th year, and find myself reflecting on the remarkable milestones achieved…

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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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