September 26, 2018

PPMD Continues Commitment to Duchenne Newborn Screening through Launch of Pilot in New York State

– PPMD Continues Commitment to U.S. Duchenne Newborn Screening Program – For the last 4 years, PPMD has been leading a national effort to build a Newborn Screening (NBS) infrastructure for Duchenne in the U.S. aimed…

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September 25, 2018

Catabasis Initiates Phase 3 PolarisDMD Clinical Trial For Edasalonexent In Duchenne

Catabasis Pharmaceuticals, Inc. announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent in Duchenne muscular dystrophy. PolarisDMD clinical trial sites across the United States will open in the coming days for enrollment of the…

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September 24, 2018

Sarepta Therapeutics Announces Clinical Hold Lifted for its Micro-dystrophin Gene Therapy Program

We are thrilled to learn that the FDA has lifted the clinical hold for Sarepta’s Duchenne micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical…

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September 18, 2018

Your Duchenne Registry Data: Researchers Find Different Mutations Have Varying Effects on Progression

As curators of The Duchenne Registry, we are frequently asking you to submit your genetic test report to the Registry. You may often wonder why we need the actual laboratory report, and why we are…

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September 17, 2018

Behavior Challenges and Duchenne: Part 1 – Encouraging Positive Behavior

Psychosocial health (also called “mental health”) includes behavior, emotional adjustment, learning/school success, and relationships. These things are very important for overall well-being and quality of life. Addressing the psychosocial needs of a person with Duchenne…

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September 17, 2018

PPMD’s Ryan Fischer Joins the Muscular Dystrophy Coordinating Committee (MDCC)

When our community first came together with our Congressional champions in 1999 to carve a path forward for our Duchenne community, among the priorities we had was for all federal agencies that impacted our community…

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September 14, 2018

2018/2019 Flu Vaccination Recommendations

The flu virus is constantly changing, with new “models” of flu appearing each year. The Center for Disease Control and Prevention (CDC) looks at the strains of influenza that have affected Australia during their flu…

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September 13, 2018

Genentech/Roche RG6206 Community Letter

  Today, Roche provided the community on their clinical development program of RG6206, an investigational anti-myostatin adnectin that is designed to block the activity of myostatin. Roche also answers questions they have received in light…

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September 12, 2018

20th Annual Mitchell’s Run Thru Rockford Another Huge Success to Help End Duchenne

On August 18th, the 20th Annual Mitchell’s Run Thru Rockford raised an incredible $85,000 to help our fight to end Duchenne. This was the most successful event to date! Thank you to Steve, Sandy, Halle,…

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September 11, 2018

Natural disaster resources for families living with Duchenne

We are officially in the midst of hurricane season, with a major storm aimed at a large portion of the east coast later this week. To all of our PPMD Family in the path of…

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