March 12, 2021

Gaming to End Duchenne

Gaming to End Duchenne What better way to plan a DIY fundraiser than with a built-in community? Patrick Denger wanted to do something to support PPMD’s mission, so he reached out to his community of…

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March 10, 2021

Action Alert! Support Progress in the Fight Against Duchenne

Urge your House and Senate Members to Support the 2022 Duchenne Funding Request & BENEFIT Act 20 years ago, Congress held a first-ever hearing on muscular dystrophy, paving the way to the landmark MD-CARE Act…

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March 5, 2021

COVID-19 Vaccination & Duchenne: Understanding the new adenovirus-based vaccine (Janssen/J&J)

As vaccines become available to help protect against COVID-19, PPMD is here to help you find answers to questions you might have about them. Please visit PPMD’s COVID-19 Information Center for additional resources and submit…

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March 5, 2021

WATCH: Federally Funded Research Studies in Duchenne (Webinar Recordings)

Every year our community heads to Washington, D.C. as part of PPMD’s Advocacy Conference (this year, being held virtually on March 10th — and there’s still time to register!) to communicate to Congress the need for continued…

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March 4, 2021

Santhera Announces Completion of First 6-Month Period of VISION-DMD Trial with Vamorolone in Duchenne

Santhera Pharmaceuticals has reached an important milestone in the VISION-DMD study of vamorolone in people with Duchenne, conducted by partner ReveraGen Biopharma Inc. The company today announced that it has completed the last visit of…

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March 3, 2021

PPMD Awards University of Florida Wellstone Center $348,891 Grant for Duchenne Gene Therapy Research

PPMD is pleased to award the University of Florida (UF) Wellstone Muscular Dystrophy Cooperative Center a grant for $348,891. The project titled “Understanding and Improving AAV Gene Therapy for DMD” is led by H. Lee…

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February 25, 2021

FDA Grants Accelerated Approval to AMONDYS 45™ (Casimersen)

PPMD is excited to learn that the FDA has granted accelerated approval to AMONDYS 45™ (casimersen), a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to…

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February 25, 2021

Senate Duchenne Champions Reintroduce BENEFIT Act Heading into PPMD’s Virtual Advocacy Conference

When people think of dynamic duos and classic pairings, they probably think of Batman and Robin, Fred and Ginger, or Oreos and milk. For those of us in the Duchenne and rare disease community, we…

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February 22, 2021

Golf Outings: The Perfect Socially-Distanced DIY Event!

Jared Sweet Golf Outing: The Perfect Socially-Distanced DIY Event! Sue and Jeff Sweet have held the Jared Sweet Golf Outing for 14 years. Thankfully they were able to alter their 2020 event and make it…

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February 22, 2021

New Resource! Common Labs and What They Mean in Duchenne

A variety of laboratory tests are often obtained in the assessment and treatment of Duchenne, and those laboratory tests can be incredibly confusing and variable in nature. As such, PPMD has developed a new resource…

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