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Tiffany Mulloy

Race Athlete Spotlight: Tiffany Mulloy

Tiffany Mulloy recently participated in the Cincinnati Flying Pig Marathon Weekend for the 2nd year in a row, in honor of her son Allan and all those living with Duchenne. Tiffany raised more than $11,000…

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PPMD resubmits RUSP nomination

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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Summer fun

Summer Fun: Accessible Activities for All Ages and Interests

As the school year wraps up, we are all looking forward to a fun-filled summer. It can be overwhelming to plan outings and activities considering accessibility and necessary accommodations to keep everyone safe. Along…

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Sarepta Announces PDUFA Extension and Update for the FDA’s Review of SRP-9001

Sarepta Therapeutics announced today that they received an update from the FDA on the Biologics License Application (BLA) for SRP-9001. The PDUFA date reportedly has been extended to June 22, 2023. …

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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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A Community United: Thank You for Raising Your Voice During the SRP-9001 Advisory Committee

Today was a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy intended to treat Duchenne. This meeting was held as part of the regulatory review process for…

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WATCH: Edgewise — A Novel Approach Designed to Protect Muscle in Becker (Webinar Recording)

PPMD was joined by Edgewise Therapeutics for a community webinar on May 3, 2023 to discuss the company’s novel approach designed to protect muscle in individuals living with Becker muscular dystrophy. The webinar highlighted EDG-5506,…

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May 12: Advisory Committee Meeting for SRP-9001 – Live Stream Information & Meeting Materials Released

The FDA has shared live stream access information for the May 12th Advisory Committee for SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics’s investigational gene therapy, which has applied for FDA approval through the Accelerated Approval pathway with…

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What You Need To Know Ahead of the SRP-9001 Advisory Committee on Friday, May 12

This Friday, May 12th, will be a historic day for the Duchenne community. It will be the day of the first ever FDA Advisory Committee meeting for a gene therapy specifically to treat Duchenne. PPMD…

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PPMD Submits Findings from New Gene Therapy Preference Study to FDA for SRP-9001 Advisory Committee

May 12 will be a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy specifically intended to treat Duchenne. PPMD, our community partners, and many others have long…

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