December 15, 2022

Give Today to help my Heart Grow Stronger

I was diagnosed with Duchenne muscular dystrophy at three years old. It was the late 90’s and PPMD was just beginning to grow as an organization. We found Pat Furlong and through her were connected…

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December 14, 2022

Back in Washington, DC! PPMD’s Advocacy Conference Returns March 5-7

Want to be part of history? Want to contribute to future progress in understanding, diagnosing, and treating Duchenne? Register today to join PPMD’s 2023 Advocacy Conference, March 5-7th, in Washington, DC! We are excited to announce…

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December 12, 2022

The Duchenne Registry Celebrates 15 Years of Data Collection

The PPMD team is incredibly grateful for the families who have shared their Duchenne and Becker experience with The Duchenne Registry over the years. It is hard to believe that the first families joined the…

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December 8, 2022

Your Gift Today Funds ACTION

As a cardiologist, I think about the heart day and night. It is central and fundamental to all of us. We must protect and preserve the heart so that individuals with Duchenne or Becker can…

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December 6, 2022

Complete Your Registry Surveys and You Could Win Your Choice of a $150 Holiday Gift Card!

It’s the Holiday Season and we are thrilled to have another great raffle for families who participate in The Duchenne Registry! This December, the Registry will be giving away TWO $150 e-gift cards to the…

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December 5, 2022

Solid Biosciences Announces Completion of AavantiBio Acquisition & Update on Duchenne Pipeline Program

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have completed the acquisition of AavantiBio, a gene therapy company. The combined company will focus on advancing a…

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November 30, 2022

Race Athlete Spotlight: Jason Hendry

Jason Hendry is gearing up for his fourth race at the 2023 Walt Disney World Marathon® Weekend presented by State Farm. Meet Jason Why did you choose to join the Race to End Duchenne team?…

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November 28, 2022

Sarepta Announces Biologics License Application (BLA) Accepted For Filing by the FDA for SRP-9001

PPMD is thrilled to learn that the FDA has accepted for filing and granted priority review for the Biologics License Application (BLA) for Sarepta’s gene therapy product, SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant…

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November 23, 2022

This Thanksgiving I am Grateful for You.

As I prepare for Thanksgiving, I run through a quick inventory in my head. I’ve got turkey and stuffing, a pumpkin pie with lots of whipped cream, and of course, Diet Coke. Most importantly, family…

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November 21, 2022

PTC Shares Community Letter Updating on Activities To Bring Ataluren (Translarna) To US Patients

PTC Therapeutics has shared a community letter updating on the company’s activities to bring ataluren (Translarna) to US patients with nonsense mutations. PPMD will host a community webinar with PTC on Friday, December 9th at…

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