February 27, 2024

Breaking News: Rare Disease Day Congressional Hearing to Address the BENEFIT Act

On Thursday, February 29th, Rare Disease Day, the House Energy and Commerce Committee will hold a congressional hearing, titled “Legislative Proposals to Support Patients with Rare Diseases” at 10:00 am ET. This hearing will review…

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February 23, 2024

Watch: Overview of WVE-N531 and the FORWARD-53 Clinical Trial with Wave Life Sciences (Webinar Recording)

Wave Life Sciences recently joined PPMD for a community webinar to share an update on the company’s FORWARD-53 study. Wave provided an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular…

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February 23, 2024

Revised Community-Led Duchenne Guidance Published: Developing Potential Treatments for the Entire Spectrum of Disease

Today PPMD is pleased to share that the revised community-led Duchenne Guidance has been published in the Journal of Neuromuscular Diseases! This Community Draft Guidance was submitted to the FDA at the end of 2022….

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February 21, 2024

PPMD Announces Pediatric Certified Duchenne Care Center at Penn State Health Children’s Hospital

Today PPMD announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Penn State Health Children’s Hospital in Hershey, Pennsylvania. This designation marks a significant step…

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February 16, 2024

FDA Accepts Efficacy Supplement to Expand ELEVIDYS Indication

PPMD is excited to learn that the U.S. Food and Drug Administration (FDA) has accepted and filed Sarepta Therapeutics’ efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS. The efficacy supplement aims to ​​expand…

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February 14, 2024

WATCH: PTC Therapeutics — EMFLAZA® Landscape in 2024 (Webinar Recording)

PPMD was recently joined by the team at PTC Therapeutics to discuss upcoming changes and continuing community support around EMFLAZA (deflazacort). EMFLAZA came on the U.S. market in 2017 as the first FDA-approved steroid specifically…

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February 13, 2024

PPMD Provides $500,000 in Funding to Kinea Bio Through PPMD Venture Pathways Program to Support Next-Gen Midi-Dystrophin Gene Therapy Development

PPMD is excited to announce that the organization has provided $500,000 in funding to Kinea Bio, Inc. (Kinea) through PPMD Venture Pathways, the organization’s venture philanthropy program which provides industry funding to accelerate therapeutic development….

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February 9, 2024

Update From PPMD’s 2024 PPMD Duchenne Healthcare Professionals Summit

PPMD recently hosted our fifth in-person Duchenne Healthcare Professionals Summit in Sanibel, FL. Over the course of three impactful days, we were joined by around 300 healthcare professionals, researchers, and industry partners to collaborate, connect,…

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February 8, 2024

Watch: Dyne Therapeutics — Advancing DYNE-251, an Investigational Medicine for DMD (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a community webinar to present initial clinical data from the DELIVER trial of DYNE-251, a next-generation exon skipping investigational product, in individuals with Duchenne who are amenable to exon…

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February 8, 2024

REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial

REGENXBIO Inc. has announced that enrollment has completed at dose level 2 of the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne. RGX-202 utilizes a…

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