January 12, 2021

WATCH: PGTME Hosts Gene Therapy Lecture Series

As the Duchenne community enters the era of gene therapy, many new concepts, terms and issues arise, including those related to research design, patient autonomy, sibling and family involvement, fairness in access and the quality…

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January 12, 2021

Join PPMD’s 20th Anniversary of Advocacy in Action

2021 marks the 20th Anniversary of the MD-CARE Act, landmark legislation that launched coordinated federal efforts to confront Duchenne and Becker muscular dystrophy through research, public health, and quality care initiatives. This congressionally directed federal…

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January 7, 2021

Sarepta Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001 Gene Therapy

Sarepta Therapeutics today announced top-line results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)…

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January 7, 2021

Pfizer Doses First Participant in Phase 3 Study for Duchenne Investigational Gene Therapy

More exciting news for our community! Pfizer today announced that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of their investigational AAV9 mini-dystrophin gene…

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January 7, 2021

PPMD Announces 2021 Certified Duchenne Care Center Inter-Institutional Collaboration Grant Program

Parent Project Muscular Dystrophy (PPMD) is pleased to announce the opportunity for members of our Certified Duchenne Care Center network to apply for a grant to support an inter-institutional collaborative project to focus on advancing…

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January 5, 2021

REGENXBIO Announces New Gene Therapy Program for Duchenne Muscular Dystrophy

PPMD is incredibly excited to share REGENXBIO’s announcement of their new program for Duchenne muscular dystrophy. REGENXBIO is seeking to treat Duchenne through a gene therapy approach delivering a novel microdystrophin transgene. Their gene therapy…

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January 5, 2021

Because of You, We Can Unleash Breakthroughs

Thanks to your generosity, I am proud to announce that we reached our goal of $400,000 to fund PPMD’s Protein Mapping Project, making the formal launch of our Biomarkers Initiative a success! I am thankful…

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December 31, 2020

Give before midnight. Unlock breakthroughs.

I can’t remember ever looking forward to a new year as much as I am looking forward to 2021. Tonight, as we ring in this hopefully brighter and better new year, I will take a…

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December 30, 2020

Tomorrow is the last chance to double your gift

We’re almost there. Tomorrow at midnight we will ring in a new year, one we all hope will bring a slow return to normalcy, to being together again, and to embracing our loved ones. This…

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December 28, 2020

PPMD Advocates Secure Critical Congressional Funding for Duchenne Priorities in Year-End Package

Thanks to your advocacy at our annual spring Advocacy Conference supporting congressional funding of Duchenne research and care priorities, Congress has continued to fund federal agencies for this work and the President has signed off…

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