Celebrating Breaking News in DMD Treatment

FDA Approves Sarepta Therapeutics’ ELEVIDYS [Muscular Dystrophy Association]( [Muscular Dystrophy Association Celebrates FDA Approval of ELEVIDYS]( MDA Celebrates the Groundbreaking Approval of ELEVIDYS for Treatment of Duchenne Muscular Dystrophy The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of ELEVIDYS, for the treatment of Duchenne muscular dystrophy (DMD) in young boys who have a confirmed mutation of the DMD gene. ELEVIDYS is the first gene therapy for DMD, and is a one-time treatment designed to treat the underlying cause of the rare and fatal genetic muscle disease. Initially, it will be for use in patients aged four to five years old. In the last six years, four targeted drugs have been approved by the FDA to treat DMD: Exondys 51, Vyondys 53, and Amondys 45 from Sarepta Therapeutics, and Viltepso from NS Pharma. [Read the Full Announcement]( At the Forefront of DMD and Gene Therapy Advancements MDA has been at the forefront of advancements in DMD for over seven decades, investing almost $227 million in research for DMD and Becker muscular dystrophy (BMD). In addition, MDA has invested over $125M in the development of gene therapy for neuromuscular diseases and has recently created the [MDA Gene Therapy Support Network (GTx)]( to provide resources and guidance for clinicians and families. Click below to learn more about and watch MDA’s 70+ year history of neuromuscular disease research and advancements in treatment and gene therapy development. [Watch Now]( You Can Help Make This Possible “This is really happening. We went from hope to actually seeing an amazing gene therapy. Being able to buy 5 to 10 years for Conner is, especially for Duchenne, a miracle,” said Christopher Curran, whose 12 year old son, Conner, was diagnosed with DMD at age 4. Celebrating this groundbreaking advancement is possible because of dedicated donors who help make treatments like ELEVIDYS a reality for many like Conner. Consider [giving a gift]( to help MDA continue this crucial work for years to come. --------------------------------------------------------------- [Facebook]( [Twitter]( [Instagram]( [LinkedIn]( [YouTube]( [MDA.org]( © 2023 Muscular Dystrophy Association. All rights reserved. 161 N. Clark, Suite 3550 Chicago, IL 60601 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](