Blog

Mallinckrodt Announces First Patient Enrolled in Phase 2 Study of MNK-1411 in Duchenne Muscular Dystrophy

PPMD is excited to learn that Mallinckrodt enrolled the first patient in their Phase 2 study assessing the efficacy and safety of the investigational drug MNK-1411 (cosyntropin, also known as tetracosactide). We look forward…

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Sarepta Therapeutics Announces Clinical Hold on Micro-Dystrophin Gene Therapy Trial

This afternoon Sarepta Therapeutics, Inc. announced that the micro-dystrophin Phase 1/2a gene therapy trial was placed on clinical hold by the FDA as the result of an out-of-specification production lot. The company was notified by the Research…

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WATCH: HOPE-2 Duchenne Clinical Trial – Webinar Recording with Capricor

Capricor Therapeutics recently joined PPMD for a community webinar update to discuss the HOPE-2 trial. During the webinar, Capricor provided a background of CAP-1002 and its clinical development to date that has led to the…

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PPMD Hosts Largest Annual Conference — Presentations Available for Download!

Thank you to everyone who joined us in Scottsdale, AZ for the 2018 Annual Conference! While the extensive agenda, research presentations, break-out sessions, and interactions with industry leaders were all an incredible part of this…

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PPMD Partners with PLAE Shoes

PPMD is excited to announce a new partnership with PLAE Shoes, a leader in comfortable, stylish, hand-crafted shoes for adults and kids. PLAE is a brand many families have turned to in our community because…

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Hayes Mechanical Golf Outing Raises Over $130,000 in the Fight to End Duchenne

On June 19, 240 golfers and volunteers came out for the annual Hayes Mechanical Golf Outing in memory of Patrick and Bobby Chilcutt at the Harborside Golf Course in Chicago. The outing is a corporate…

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2018 Annual Conference Resource Round Up

PPMD’s 2018 Annual Conference Resource Fair Providers Each year we welcome specialized resource providers from around the country to interact with us at PPMD’s Annual Conference. We so appreciate the time and investment from all…

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15th Annual Jumping Jack Charity Golf Tournament Another Huge Success to Help End Duchenne

The 15th Annual Jumping Jack for Duchenne MD Golf Tournament, Wine Tasting, Dinner & Silent Auction that took place on June 18thin Westminster, CO was another huge success! The Knight Family has raised over $530,000 over…

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Summit Therapeutics Announces PhaseOut DMD Did Not Meet Primary Endpoint

We are disappointed to learn that Summit Therapeutics plc today announced that their PhaseOut DMD trial has not met its primary or secondary endpoints after 48 weeks of treatment of ezutromid in patients with Duchenne….

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Vamorolone (VBP15) Top Line Data Shows Improvements in Strength and Endurance in Boys with Duchenne

ReveraGen BioPharma, with the Cooperative International Neuromuscular Research Group (CINRG), conducted an international clinical trial of vamorolone at 11 CINRG sites in 6 countries (US, Canada, Australia, Israel, UK, and Sweden). ReveraGen reports that the…

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