August 25, 2023

WATCH: Advocating for Access: Navigating Approved Therapies in Duchenne (Webinar Recording)

Understanding and navigating insurance access can be daunting. That’s why PPMD kicked off a new webinar series, “Advocating for Access,” earlier this week with its first monthly installment on Navigating Approved Therapies in Duchenne. PPMD’s…

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August 10, 2023

PPMD’s RUSP nomination package for Duchenne passes first hurdle in federal committee vote

PPMD’s nomination package to add Duchenne to the Recommended Uniform Screening Panel (RUSP) passed the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) Nomination and Prioritization vote today! This decision marks another exciting…

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July 5, 2023

Duchenne Newborn Screening milestone—first state with universal newborn screening for Duchenne!

For more than a decade, PPMD has worked towards newborn screening (NBS) for Duchenne, including initiating a pilot study in New York State and developing a nomination package for federal review as a recommended condition…

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June 22, 2023

FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Today is an incredible milestone for the entire Duchenne community. It is a first step. But a huge step forward. Today, the FDA has granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy…

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May 31, 2023

Update on PPMD’s newborn screening efforts

For the past decade, PPMD has been leading efforts for newborn screening for Duchenne. As part of that process, PPMD, with co-sponsor Muscular Dystrophy Association (MDA), submitted a nomination package to the Recommended Uniform Screening…

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May 19, 2023

SRP-9001: Submit Your Community Perspectives to FDA

On May 12, the FDA held an Advisory Committee meeting for SRP-9001, a gene therapy intended to treat Duchenne. The Advisory Committee meeting was used by FDA to obtain independent expert advice on scientific, technical,…

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March 24, 2023

Pat Furlong Advocates in Support of Adding Duchenne to the Ohio Newborn Screening Panel

On March 23, PPMD’s Founding President & CEO Pat Furlong addressed the Ohio House Finance Subcommittee on Health and Human Services in support of legislation that would add Duchenne to the Ohio Newborn Screening Panel….

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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 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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