Blog

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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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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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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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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February 17, 2021

Duchenne UK and PPMD Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK are excited to announce Professor Kanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of our Joint Research Grant Call of 2020. The…

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February 17, 2021

New Resource! Safe & Unsafe Medications in Duchenne

Many years ago, PPMD developed a one-page resource of safe and unsafe medications in Duchenne. While many of those medications are now discussed in various related sections of our website, we recognize the need for…

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