September 6, 2022

Pat Furlong: My Story as a Mom, Finding the Power of ‘Us’ #WomenAndDuchenne

World Duchenne Awareness Day 2022: #WomenAndDuchenne Each year, World Duchenne Awareness Day highlights a special theme for further education. This year that theme is Women & Duchenne to raise awareness about females living with ‘dystrophinopathy’ (the spectrum of muscle…

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September 6, 2022

Mariana: My Story as a Carrier with Cardiac Concerns #WomenAndDuchenne

World Duchenne Awareness Day 2022: #WomenAndDuchenne Each year, World Duchenne Awareness Day highlights a special theme for further education. This year that theme is Women & Duchenne to raise awareness about females living with ‘dystrophinopathy’ (the spectrum of muscle…

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September 6, 2022

Deb: My Story as a Mom to Daughters Living with Duchenne #WomenAndDuchenne

World Duchenne Awareness Day 2022: #WomenAndDuchenne Each year, World Duchenne Awareness Day highlights a special theme for further education. This year that theme is Women & Duchenne to raise awareness about females living with ‘dystrophinopathy’ (the spectrum of muscle…

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September 6, 2022

Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051

Sarepta Therapeutics has announced that the FDA has lifted the clinical hold on SRP-5051 (vesleteplirsen) following a serious adverse event of hypomagnesemia (low magnesium). SRP-5051 is Sarepta’s next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat…

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September 2, 2022

WATCH: Casimir’s Duchenne Video Assessment (DVA) ARISE Study (Webinar Recording)

Casimir joined PPMD for a community webinar on Wednesday, August 31, 2022 to discuss how and why video assessments may help the Duchenne community by capturing quality of movement data in clinical trials. Casimir also…

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September 1, 2022

Pfizer Shares Update on DAYLIGHT Phase 2 Gene Therapy Trial

Pfizer has issued a community letter announcing that its Phase 2 DAYLIGHT clinical trial evaluating the company’s investigational mini-dystrophin gene therapy product (fordadistrogene movaparvovec) has opened a first trial site in Australia. Pfizer shares that…

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August 31, 2022

Gear Up For Coach To Cure MD Game Day!

Gear Up For Coach To Cure MD Game Day Our 15th Annual Coach To Cure MD is just under a month away! Although game day kick off isn’t until September 24th, there is still time…

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August 24, 2022

Sign up today! Virtual Series-Global Walk 4 Duchenne

Sign up today for the Race to End Duchenne Virtual Series – Global Walk 4 Duchenne September 1 – September 30, 2022! Join the movement as we gather to bring awareness to Duchenne muscular dystrophy…

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August 22, 2022

Santhera & ReveraGen Announce First Participant Dosed in Phase 2 Pilot Study with Vamorolone in Becker

Today Santhera and ReveraGen announced they have dosed the first participant in their Phase 2 study with Vamorolone for individuals with Becker muscular dystrophy. The small pilot study was funded through an FDA grant and…

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August 22, 2022

WATCH: Dyne Therapeutics Update on DYNE-251 (Webinar Recording)

Dyne Therapeutics joined PPMD for a community webinar on Wednesday, July 20, 2022. Dyne provided an update on the recent U.S. Food and Drug Administration (FDA) announcement lifting the DYNE-251 clinical hold and clearing the…

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