US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

Sign Up for MDA News & Updates

Dr. Donald S. Wood, MDA President and CEO.

A message from Donald S. Wood, PhD, President and CEO

Rare neuromuscular diseases stand as some of the most challenging puzzles in science and medicine today. The rare nature of progressive muscle disease like muscular dystrophies, spinal muscular atrophy, ALS, and Pompe, coupled with corresponding gaps in public awareness and scientific investment, can present obstacles to research and access to treatments. But at the Muscular Dystrophy Association (MDA), thanks to your support, we're tackling these challenges head-on and we’re seeing results!

Our work has led to historic advancements in genetic research, improved diagnostic methods, multidisciplinary care, and the development of groundbreaking therapies that once seemed beyond reach. These scientific discoveries have wide-reaching applications for other more commonplace diseases and conditions, affecting millions of people across the globe.

Together, we can surge past the limits of yesterday's understanding and treatments. It’s your commitment, involvement, and support that are the forces that drive us forward. Thank you for your dedication to people living with neuromuscular diseases and to MDA’s mission.

Mission Statement

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. 

MDA's mission is to empower the people we serve to live longer, more independent lives.

  • Innovations in Science

    We are accelerating the delivery of treatments and cures.

    Explore the Science
  • Innovations in Care

    We provide services that help people affected by neuromuscular disease.

    Experience the Care

The freedom to walk, to talk, to run and play. To laugh, to hug, to eat — even breathe.

Each day these freedoms are taken away from kids and adults with muscular dystrophy, ALS and related diseases that weaken muscle strength and limit mobility. Together we can change that.

About Neuromuscular Diseases

Get Involved

There are many ways to get involved with MDA. Whether you’re looking to create a fundraiser, host a game night, volunteer at a camp, run a marathon, or advocate for the cause, the ways to get involved are endless.

Ways to Get Involved

Latest Stories

For the latest happenings in the MDA community and to read inspiring stories, browse the MDA blog.

Visit Our Blog

Press Releases

Muscular Dystrophy Association Announces Expanded US FDA Approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy Patients Ages 4 and Above
Milestone Expanded Approval Brings Hope for Families Living with Duchenne Muscular Dystrophy
Muscular Dystrophy Association Announces Collaborative Infrastructure Research Grant with Support from RYR-1 Foundation for Nearly $300,000 to Joshua Todd, PhD at NIH for RYR...
Dutch Bros raises $2.5M for the Muscular Dystrophy Association
See Recent News

Meet Our Partners

These partners are empowering lives through science and care for MDA families.

Two sponsor logos are shown, IAFF, and CITGO.
Meet Them All

Join Us

Sign up to receive email updates on volunteer opportunities and the impact you’re making for families.

MDA Resource Center: We’re Here For You

Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 1-833-ASK-MDA1 (1-833-275-6321). If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA programs are only available in the U.S.

Request Information