BREAKING NEWS: Expanded US FDA Approval of ELEVIDYS Gene Therapy

for Duchenne Muscular Dystrophy Patients Ages 4 and Above [Muscular Dystrophy Association]( [Expanded US FDA Approval of ELEVIDYS Gene Therapy]( Muscular Dystrophy Association Applauds Expanded FDA Approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy The Muscular Dystrophy Association (MDA) celebrates the U.S. Food and Drug Administration’s expanded approval of ELEVIDYS (delandistrogene moxeparvovec-rokl). The efficacy supplement approval will expand the labeled indication of ELEVIDYS to include boys and men with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age. The FDA granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients, confirming the functional benefits of the treatment. Developed by Sarepta Therapeutics, ELEVIDYS targets the genetic root cause of Duchenne muscular dystrophy (DMD) and works by introducing an engineered gene that codes for a shortened version of dystrophin, referred to as ELEVIDYS micro-dystrophin into muscle cells. The FDA granted accelerated approval for boys ages 4 and 5 in [June of 2023](. [Read the Full Announcement]( “The FDA’s expanded approval for ELEVIDYS marks a pivotal moment for the community, offering renewed hope and tangible progress in our fight against DMD,” said Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association. "At the Muscular Dystrophy Association, we continue our legacy and impact with innovative scientific and clinical research to propel treatments and therapies forward for the families we serve.” “By expanding the label beyond current age groups, the FDA is exercising flexibility in its approach to a rare disease with high unmet need. Many older boys and young men urgently need access to new therapies that could alter the disease's progression. By broadening the indication, hope can be offered to more families and improve the quality of life for a larger segment of the DMD community”, said Paul Melmeyer, EVP, Public Policy and Advocacy, MDA. [Learn More]( “There weren’t many options when Conner was diagnosed with Duchenne muscular dystrophy nine years ago but with the MDA they’re accelerating research, and he did get gene therapy. I believe he would be in a wheelchair now full time if he hadn’t received it. Muscular Dystrophy Association was an intricate part of that process,” said Jessica Curran, MDA family member. MDA remains steadfast in its mission to accelerate the development of treatments and cures for neuromuscular diseases. The organization recently announced expanded support for the families and clinical community through the MDA Gene Therapy Support Network (GTxSN), which provides guidance and support on novel gene therapy treatments, including ELEVIDYS. [Continue the Impact]( --------------------------------------------------------------- [Facebook]( [Twitter]( [Instagram]( [LinkedIn]( [YouTube]( [MDA.org]( © 2024 Muscular Dystrophy Association, Inc.. All rights reserved. 1016 W Jackson Blvd #1073 Chicago, IL 60607 1-800-572-1717 [Unsubscribe]( | [State Fundraising Notices]( The Muscular Dystrophy Association (MDA) is a qualified 501(c)(3) tax-exempt organization. [View Online]( | [Subscription Center]( | [Privacy Policy](