November 21, 2018

Giving Thanks…for YOU!

As I write this, something is inevitably overcooking in my oven, the turkey may not be defrosted, and we are running low on Diet Coke. But amidst the craziness and chaos of the holiday, I…

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November 20, 2018

Santhera Enters into Agreement to Acquire Option for Exclusive Sub-License of Vamorolone

PPMD has learned that Santhera Pharmaceuticals and Idorsia Ltd have entered into an agreement under which Santhera will acquire the option to exclusively in-license vamorolone, currently being developed by Reveragen. Idorsia currently holds the license…

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November 19, 2018

PPMD 2018 Annual Conference – Polling Report

The 2018 PPMD Annual Conference provided a unique opportunity to conduct live polling of those joining us, including those joining in person and those joining virtually from home. The following report (download) provides the data…

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November 18, 2018

WATCH: Engaging Families in Care Guideline Development – Webinar Recording

On Wednesday, November 14, PPMD hosted a webinar on patient-centeredness of recently published endocrine care guidelines for Duchenne. Researchers from RAND Health Care and PPMD shared the results of their project that developed a new online…

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November 18, 2018

Slippery Rock Celebrates Coach To Cure MD with a Race to End Duchenne .1K

Slippery Rock University’s Coach To Cure MD game earlier this month was a memorable one! During halftime, three families were welcomed on the field with open arms to have their very first annual Race to End…

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November 18, 2018

8th Annual GR .1K Raises Over $60,000 in the Fight to End Duchenne

The 8th annual GR .1K had over 1,200 racers and raised over $60,000 this year! This incredibly clever Race to End Duchenne .1K event is fun for everyone in your community – a short race,…

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November 15, 2018

WATCH: PolarisDMD – Phase 3 Clinical Trial of Edasalonexent – Webinar Recording

PPMD and Catabasis as hosted a webinar on Wednesday, November 7 to discuss the Phase 3 PolarisDMD clinical trial studying edasalonexent in Duchenne muscular dystrophy, which is enrolling boys ages 4 to 7 (up to…

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November 13, 2018

Solid Provides Update on IGNITE DMD Clinical Trial

  Solid Biosciences Inc. today shared on a updated on the IGNITE DMD clinical trial, noting the dosing of two additional patients with SGT-001 gene transfer and the company’s plan to report preliminary results from IGNITE…

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November 6, 2018

PPMD Designates Children’s Hospital of Wisconsin a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named the Neuromuscular Disorders Program at Children’s Hospital of Wisconsin to be the 21st clinic to be named a Certified Duchenne Care Center PPMD’s Certified Duchenne Care Center Program,…

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November 5, 2018

Calling All Adults with Duchenne & Becker MD: Join the 2019 PPMD Adult Advisory Committee!

Been looking for a way to engage with the community a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts? Interested in serving as a…

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