December 20, 2018

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Golodirsen (SRP-4053)

PPMD is excited to learn that Sarepta Therapeutics, Inc.  announced that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat…

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December 19, 2018

PPMD Awards $329,000 Grant to Binghamton University to Examine Role of Immune System on Dystrophin Restoration

PPMD is excited to award Dr. Kanneboyina Nagaraju and his team at Binghamton University’s School of Pharmacy and Pharmaceutical Sciences a $329,000 grant to continue his work examining the immune response to the production of…

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December 18, 2018

A strong heart — is more time with my family

I hope this note finds you and your family enjoying the holiday season! I am a 41-year-old with Becker muscular dystrophy. Until recently, I served on PPMD’s Adult Advisory Committee (PAAC), and I’m extraordinarily proud…

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December 18, 2018

Keep hearts strong — all gifts doubled!

If you’re part of the Duchenne community, you have likely seen parents share updates from their child’s cardiac appointment. The anxiety of these appointments can be incredible — stability at a doctor’s appointment when you…

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December 17, 2018

End Duchenne Tour Recap: Cincinnati, OH

PPMD headed to Cincinnati, OH for our End Duchenne Tour stop on November 17, 2018. Many families and members of the Duchenne community came together to learn about the latest information and resources for Duchenne families. PPMD…

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December 13, 2018

ICER Announces the Review of Duchenne Products – What Does This Mean?

Thanks to decades of advocacy, funding and innovation, our healthcare landscape is rapidly changing. As new therapies emerge into the marketplace, patients, providers, and payers are faced with challenges as to how to discern which…

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December 13, 2018

PPMD Announces Partnership with THREAD to Enhance Current Duchenne Registry Platform

PPMD is excited to announce our new partnership with THREAD — a company specializing in remote/virtual patient research via their platform — to enhance the patient engagement and care management tools for The Duchenne Registry. Building…

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December 12, 2018

PPMD Hosts Inflammation Meeting

Steroids. If you are part of this community, you probably have strong feelings about steroids in Duchenne – feelings that can vary in range on any given day. But steroids are standard of care according…

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December 12, 2018

WATCH: The Heart of PPMD’s Research – Webinar Recording

ICYMI: Yesterday, I had the privilege of hosting an in depth look at PPMD’s cardiac research priorities in Duchenne for 2019. If you were unable to join us live, I hope you will take a…

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December 10, 2018

Your gift to critical cardiac research will be matched!

When PPMD started in 1994, research to treat Duchenne muscular dystrophy was virtually nonexistent. Nearly 25 years later, we have a clinical trial pipeline full of possibility with over 25 active trials, 30+ companies, and…

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