January 8, 2019

Meeting Update: Inflammation in Duchenne

Last month, PPMD convened a day and half meeting – Inflammation in Duchenne: Current and New Treatment Strategies – to bring academic and clinical researchers together to discuss advancements that could be made by treating…

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January 3, 2019

Wave Life Sciences Duchenne Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program

Wave Life Sciences announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne clinical program has been selected for the U.S. Food and Drug Administration (FDA) pilot program for complex innovative…

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January 3, 2019

Catabasis Phase 3 PolarisDMD Trial for Edasalonexent Update & Additional Trial Sites Open for Enrollment

Catabasis Pharmaceuticals shared an update on clinical trial sites in the United States and recent international clinical trial application approvals to perform the Phase 3 PolarisDMD trial for edasalonexent (CAT-1004) in Duchenne. PolarisDMD clinical trial…

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January 3, 2019

Because of your heart — WE DID IT!

For the last few weeks, PPMD has asked you to consider supporting the expansion of our Cardiac Initiative so that we can ensure potential Duchenne treatments support heart health. We set our most ambitious fundraising…

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December 28, 2018

We’re fighting to save the heart — join us

The Friar family is made up of fighters. We’re very nice people, don’t get me wrong! But when faced with adversity or challenges, we fight back. As a family. When our oldest son Kevin was…

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December 25, 2018

The heart of ending Duchenne

Just more than a year ago, I knew little about Duchenne muscular dystrophy and had never heard of PPMD. So much has changed since the day when our then 4-year-old grandson, Henry, was diagnosed. Today,…

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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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