March 18, 2025

A Safety Update on ELEVIDYS

We are deeply saddened by the loss of a young member of our community following treatment with ELEVIDYS. According to Sarepta, the individual with Duchenne received ELEVIDYS and later suffered from complications of acute liver…

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January 27, 2025

Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS

Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s Phase 3 clinical study of ELEVIDYS. ELEVIDYS is a micro-dystrophin gene therapy used for the treatment of individuals aged four…

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July 24, 2024

Navigating the Approved Therapy Landscape

The approved therapy landscape has changed drastically in Duchenne, especially over the last year. With multiple newly approved therapies comes a lot of questions about how to navigate these drugs: when they are prescribed; who…

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June 20, 2024

FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older

PPMD is excited to share that the FDA has made the decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy used to treat people with Duchenne muscular dystrophy, to include individuals at…

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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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December 22, 2023

Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label

Sarepta Therapeutics, Inc. today announced the submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS to expand its labeled indication. The company also shared that it completed the Phase 3 EMBARK…

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December 21, 2023

PPMD & Community Partners Urge Timely, Transparent FDA Review of EMBARK Data

Today, PPMD joined the Best Day Ever Foundation, CureDuchenne, the Jett Foundation, the Little Hercules Foundation, The Muscular Dystrophy Family Foundation, and Team Joseph in sending a letter to the Federal Drug Administration (FDA) regarding…

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October 30, 2023

Sarepta Therapeutics Announces Topline Results from Phase 3 EMBARK Study of ELEVIDYS Gene Therapy

Sarepta Therapeutics has announced Phase 3 topline results of their EMBARK study with ELEVIDYS, a micro-dystrophin gene therapy product currently approved for patients aged 4-5 living with Duchenne. Sarepta shares that while EMBARK did not…

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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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July 14, 2023

WATCH: ELEVIDYS: What This Means for You and Your Family (Webinar Recording)

In case you missed it, PPMD was joined by Drs. Russ Butterfield of University of Utah and Sue Apkon of Colorado Children’s earlier this week to talk through some of the common questions around the…

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