January 6, 2022

REGENXBIO Announces FDA Clearance of IND for Clinical Trial of RGX-202 Gene Therapy Candidate for Duchenne

PPMD is excited to share that REGENXBIO has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for RGX-202, a micro-dystrophin gene therapy product. With this clearance REGENXBIO…

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January 5, 2022

Edgewise Therapeutics Announces Positive Topline Results From EDG-5506 Phase 1b Trial in Adults With Becker

Edgewise Therapeutics today announced positive topline results from the Phase 1b clinical trial of EDG-5506 in Becker muscular dystrophy. EDG-5506 is an oral small molecule muscle stabilizer which seeks to protect the muscle from injuries…

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January 5, 2022

Sarepta Therapeutics Shares MOMENTUM, Study SRP-5051-201, Update

Sarepta Therapeutics has shared an update that SRP-5051-201, the MOMENTUM study (NCT04004065), is now recruiting Part B. MOMENTUM is a Phase 2, two-part, multiple-ascending-dose study of SRP-5051 for dose determination, then dose expansion, in patients…

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January 4, 2022

Thank YOU for Joining the Power of Us!

I am grateful for your generosity this year and excited to announce that we have reached our goal of providing supplemental funding to the Wellstone Muscular Dystrophy Cooperative Research Centers. The Power of Us is…

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December 30, 2021

Tomorrow is the last chance to double your gift

I can’t believe it’s almost the beginning of a new year. In about a week, I will lace up my sneakers once again and join my chosen family for another Walt Disney World Marathon Weekend….

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December 26, 2021

Double the Impact of Your Gift Today!

Once Duchenne enters your life, you learn to be resilient even if it is against your wishes. You learn to come together to get through the tough times, even if it is with a community…

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December 20, 2021

PPMD Signs Onto Letter to Congress in Support of Nomination for FDA Commissioner

PPMD joins over 50 organizations in a letter to Congress led by the EveryLife Foundation for Rare Diseases in support of President Biden’s nomination of Rob Califf for FDA Commissioner. Dr. Califf served as FDA…

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December 20, 2021

Update on Pfizer’s Phase 1B Open-Label Mini-Dystrophin Gene Therapy Trial for Duchenne

Today we learned that there has been a devastating loss of a young man that was participating in Pfizer’s Phase 1B Open-Label study. This study is evaluating PF- 06939926 which is Pfizer’s AAV9 Gene Therapy…

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December 17, 2021

Gene Editing Therapy Start-up Secures $3.4 Million Grant for Duchenne Research

MyoGene Bio has received a $3.4 million grant from the California Institute of Regenerative Medicine to advance its pre-clinical gene-editing studies in Duchenne, thanks to support from Parent Project Muscular Dystrophy (PPMD), Duchenne UK, and…

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December 15, 2021

Help PPMD raise $400,000 to invest in the future of Duchenne research

I am humbled to be working in Duchenne research, where patients and families have been the relentless driving force behind the progress we’ve made so far. We have seen the Power of Us realized, as…

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