December 15, 2021

Second Chance — Win a NEW Nintendo Switch OLED!

THANK YOU, Duchenne Registry participants! As you might have heard, The Duchenne Registry was thrilled to recently give away a Nintendo Switch OLED to a Duchenne Registry participant who completed all of their surveys. The…

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December 15, 2021

Edgewise Initiates ARCH Follow-On Open Label Becker Study; Will Report Phase 1b Topline Results on January 5

Edgewise Therapeutics has announced the initiation of the ARCH open label study of EDG-5506 in Becker muscular dystrophy, which will enroll participants from its Phase 1b clinical trial as well as new participants with Becker….

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December 7, 2021

Join the Power of Us with your gift today

Preparing to begin a new year, I always become a little nostalgic. I think about the year ahead of me and about the many years that have passed. This year, I can’t help but think…

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December 6, 2021

PPMD’s Drug Development Roundtable Meets to Explore Utilization of External Controls to Accelerate Research

At the heart of PPMD’s mission to end Duchenne lies our unwavering commitment to accelerate research. As part of our comprehensive research strategy, this includes a focus on enhancing the drug development process to ensure…

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December 3, 2021

Updating the Community-led Duchenne Guidance for FDA – The Power of Us!

The update to the Community-led Duchenne Guidance for FDA is well underway and on track to be submitted to the agency in the beginning of 2022. As you know, in 2014, we came together as…

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December 3, 2021

Dyne Therapeutics Announced Submission of IND Application to Initiate Clinical Trial of DYNE-251

Dyne Therapeutics, Inc. announced yesterday the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy…

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December 1, 2021

PPMD Hosts “Dystrophinopathy and the Brain” Meeting in New York City

“Is my child happy?” “Do they have friends?” “Are they learning as easily and as quickly as the other students in their class – are they keeping up?” “Can I parent this child?” “Why are…

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November 29, 2021

Support PPMD Tomorrow, on #GivingTuesday

#GivingTuesday is a global generosity movement, bringing together the power of people and organizations to transform their communities and their world. 2021 has once again highlighted  the power of our Duchenne community. Although we came…

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November 24, 2021

This Thanksgiving I am grateful for you

The turkey is prepped, and we are ready to turn on the oven bright and early tomorrow. My emotions are always heightened  during the holidays, but this year it’s even more palpable. The holidays are…

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November 23, 2021

Santhera and ReveraGen Announce Positive Topline Results with Vamorolone after Completion of the VISION-DMD Study

PPMD is pleased to share the announcement from Santhera Pharmaceuticals and ReveraGen BioPharma, Inc., of new topline results after completion of the VISION-DMD study at week 48. VISION-DMD was a pivotal double-blind Phase 2b study…

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