To Patients

Therapeutic Approach

Restoring or Replacing Dystrophin

Gene therapy for Duchenne is centered on the goal of successfully introducing into a muscle cell the correct genetic code, or recipe, necessary to make the dystrophin protein. Because dystrophin is such a large protein, smaller versions (referred to as micro-dystrophins) are inserted into the delivery vehicle. Viral delivery using the adeno-associated virus (AAV) harnesses the virus’s natural ability to deposit genetic material right to the muscle cell nucleus. The result of this viral “infection” would be the successful recoding of each muscle cell in the patient’s body so that a smaller but functional dystrophin protein could be made.


ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD). This indication is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle observed in patients treated with ELEVIDYS. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).



This program is sponsored by Sarepta Therapeutics and has been partially funded by Parent Project Muscular Dystrophy.

Related Studies

(ENVISION) A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
EMBARK – A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants With Duchenne Muscular Dystrophy
ENDEAVOR - A phase 1 clinical trial using commercial grade product

Media Library

JULY 2023

ELEVIDYS and Genetic Variants

PPMD's Niki Armstrong, MS, CGC, discusses the ELEVIDYS label and genetic variants.
JULY 2023

Webinar: ELEVIDYS Approval - What this means for you and your family

PPMD was joined by Drs. Russ Butterfield of University of Utah and Sue Apkon of Colorado Children’s to talk through some of the common questions around the recent FDA accelerated approval of ELEVIDYS. Drs. Butterfield and Apkon provided an overview of how gene therapy works, eligibility criteria listed in the ELEVIDYS label, risks and benefits, as well as logistics for accessing therapy safely.

Webinar: SRP-9001 Data Update from Studies 101, 102, and 103 (August 2022)

On August 24, 2022, PPMD was joined by Sarepta Therapeutics for a community webinar to present a SRP-9001 gene therapy data update from Studies 101, 102, and 103, following their presentation at a medical conference in July.

Webinar: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (October 2021)

PPMD was joined by Sarepta Therapeutics for a webinar on October 13, 2021 for an update on the company's EMBARK clinical trial (SRP-9001-301).
JUNE 2021

Webinar: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update

Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar update on June 2, 2021. Sarepta was invited to present recent updates from their ongoing clinical trials, 9001-102 and 9001-103.

Webinar: SRP-9001 Gene Therapy Path Forward

PPMD and Sarepta Therapeutics hosted a webinar on November 24, 2020 for a discussion about the SRP-9001 gene therapy development program.
* This webinar recording contains edits to content requested by the company.
JUNE 2019

Sarepta Presents at the PPMD 2019 Annual Conference

JUNE 2018

Jerry Mendell, MD (Nationwide Children’s) Presents at the PPMD 2018 Annual Conference


Webinar: Understanding Gene Therapy, Part 2 - Nationwide Children's Hospital

As part of our ongoing series on Understanding Gene Therapy, Dr. Jerry Mendell of Nationwide Children’s Hospital joined PPMD for a webinar on September 6, 2017 to discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines. This webinar is the second in our series on Gene Therapy where we are bring leaders in gene therapy technology to you to discuss their latest projects and provide an update on this incredibly exciting and expanding field.

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