March 16, 2023

Sarepta Therapeutics Announces Advisory Committee Meeting will be Held for SRP-9001

Sarepta Therapeutics today shared an update regarding SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, for which a Biologics License Application (BLA) is currently under review by the U.S. Food and Drug…

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March 6, 2023

Action Alert! Urge Congress to Support Progress in the Fight to End Duchenne

Today, for the first time since 2020, PPMD Advocates are back in person in Washington, DC to make their voices heard in the fight to end Duchenne! Over the next two days, more than 130…

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February 28, 2023

Duchenne Champions Reintroduce BENEFIT Act Heading into PPMD’s 2023 Advocacy Conference

PPMD applauds Senators Roger Wicker and Amy Klobuchar and Representatives Doris Matsui and Brad Wenstrup for the reintroduction of the BENEFIT Act, bipartisan legislation conceived by PPMD in collaboration with Congressional Champions that will help…

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February 28, 2023

Race Athlete Spotlight: Anita Mullick

Joining the Race to End Duchenne Virtual Series can be done from anywhere!  Anita supports PPMD all the way from Australia.  Our next Virtual Series is coming this May! Meet Anita Why did you choose…

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February 24, 2023

Meet the 2023 PPMD Adult Advisory Committee (PAAC)

PPMD’s Adult Advisory Committee (PAAC) welcomes 10 new members this year, bringing the group to a total of 36 individuals living with Duchenne/Becker! We range in age from 18-63 and continue to amplify the patient…

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February 17, 2023

PPMD Announces Pediatric Certified Duchenne Care Center at Phoenix Children’s

Today Parent Project Muscular Dystrophy announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with the pediatric certification of the clinic Phoenix Children’s, one of the nation’s fastest-growing pediatric healthcare systems. This addition…

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February 16, 2023

An update about our team

I’m writing to share that Ryan Fischer is transitioning off our team next month as PPMD’s Chief Advocacy Officer. Ryan joined PPMD in 2004. During his initial interview, I mentioned that we were looking for…

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February 13, 2023

Watch: Avidity Biosciences – EXPLORE44 Clinical Trial Investigating AOC 1044 – Webinar Recording

WATCH: Avidity Biosciences – EXPLORE44 Clinical Trial Investigating AOC 1044 in Individuals Amenable to Exon 44 Skipping Avidity Biosciences recently joined PPMD for a community webinar on February 8, 2023 to discuss the EXPLORE44™ clinical…

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February 10, 2023

Update on PPMD’s Newborn Screening Efforts: ACHDNC Advises Additional Information Needed

With a robust therapeutic pipeline, regulatory infrastructure, and clinical care network and guidelines in place, PPMD has been building the infrastructure and preparing for the implementation of newborn screening for Duchenne for almost a decade….

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February 6, 2023

Race Athlete Spotlight: Lauren Casey

It takes a village in a Duchenne/Becker Family.  Lauren Casey is an aunt to two boys living with Becker muscular dystrophy and has joined the Race to End Duchenne in their honor.  She took on…

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