About Us

Muscular Dystrophy Association Announces Gene Therapy Support Network for Families Living with Neuromuscular Disease

MDA Gene Therapy Support Network offers guidance on MDA Care Centers, resources, and educational programming beginning with MDA Virtual Learning: Gene Therapy 101 webinar to be held Thursday, June 15, 4-5:30pm ET

NEW YORK, May 22, 2023 -- Muscular Dystrophy Association (MDA) announced today the launch of the MDA Gene Therapy Support Network (GTx), ahead of a pending FDA approval for the first gene therapy for Duchenne muscular dystrophy (DMD). The expanded nationally renowned MDA Resource Center offers nationwide one-on-one expert support and guidance for newly approved gene therapy treatments for people living with neuromuscular diseases (NMDs). FDA approvals for gene therapies in NMDs include treatment for spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). Families may contact MDA Resource Center Gene Therapy Specialists for information, guidance and help facilitating access to novel therapies, by phone at 1-833-ASK-MDA1 (1-833-275-6321) or via email at ResourceCenter@mdausa.org. Virtual appointments may also be scheduled at: GTx Support Specialist Meeting.

MDA GTx will include clinical networking to support ongoing clinical collaboration throughout the MDA Care Center Network, long-term family support and best practice sharing between care centers. In addition, MDA GTx will provide gene therapy resources and educational opportunities for families to inform their decision-making process.

“The Gene Therapy Support Network and the advances it represents are more than 70 years in the making; MDA pioneered the genetic research that has brought us to this exciting moment in time for families impacted by neuromuscular disease. Through MDA GTx, we will offer expert one-on-one direct support for our community to navigate the complexities of new and emerging gene therapies. MDA GTx will include clinical networking to support ongoing clinical collaboration throughout the MDA Care Center Network, long-term individual and family support and best practice sharing between our clinics. In addition, MDA GTx will launch educational resources and opportunities to build health literacy for individuals and families interested in seeking gene therapy to inform their decision-making,” said Donald S. Wood, Ph.D., President and CEO, MDA.

“It is thrilling to finally have more treatment options that can slow the progression of disease for the families we care for throughout the MDA Care Center Network. After decades of funding towards our work in scientific and clinical research, side-by-side with families, we now have even more hope with gene therapy treatments. It’s been an honor to work with my colleagues throughout the country in the MDA Care Center Network to continue the innovative work that has driven progress for the neuromuscular disease community through decades of discovery. Today, we have more tools and technology, and with gene therapy, the intention is to treat or prevent disease by administering new genetic material into affected cells using a delivery vehicle known as a vector. Genes provide instructions on how to make the proteins our bodies need. Gene therapies only target certain cells in the body, so a person's basic genetic composition is not changed, just the cells targeted by the therapy,” said Barry Byrne, M.D., Ph.D., Chief Medical Advisor, MDA and Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida.

The first in a series of educational programming features a family education event called MDA Virtual Learning: Gene Therapy 101 webinar to be held Thursday, June 15, 4-5:30pm ET with MDA Care Center Director Craig Zaidman, M.D. and Natalie Goedeker, MSN, CPNP, from Washington University in St. Louis. The event will include the discussion of the basics of gene therapy, what it is, and how it works. Attendees will receive an overview of the latest developments in gene therapy specifically for neuromuscular diseases and understand what considerations need to be taken before pursuing gene therapy treatment. Attendees will have the opportunity to ask questions during the live Q&A. This webinar will also be offered in Spanish. Registration is free (and required) for the community here. This webinar will be recorded for on-demand viewing and will be available three weeks following the live program.

“We are excited to partner with the Muscular Dystrophy Association for this Gene Therapy 101 Webinar, in which we will be discussing the background of gene therapies and relevant issues for access, administration, and follow-up care,” said Goedeker.

Muscular Dystrophy Association’s investment in gene therapy

Watch MDA’s Gene Therapy Story - Duchenne Muscular Dystrophy - here.

For more than 70 years, MDA has been committed to people living with neuromuscular disease (NMD) and is currently the largest source of research funding for NMD outside of the federal government. MDA has invested over $125 million in the development of gene therapy over the past 73 years. For decades, MDA has supported researchers who have worked to discover gene-causing mutations for neuromuscular disorders, developed and refined gene delivery tools and methods, and established protocols for safe and effective gene therapy.

Gene therapy funding provided by MDA resulted in essential milestones, including the discovery of the gene that is involved in DMD by Dr. Lou Kunkel in 1986, the development of the microdystrophin strategy in the 1990's and the first gene therapy trial for DMD in 2006. In 2019, MDA was recognized for its contributions and awarded the Sonia Skarlatos Award for Public Service by the American Society of Cell and Gene Therapy (ASCGT).

MDA’s efforts to develop this technology for application in human clinical trials provided the foundation for the first gene therapy treatment for a neuromuscular disease – a life-saving treatment for SMA – which received full approval from the FDA in 2019 for children under the age of two years.

MDA continues to lead advocacy efforts for accelerated access to therapies and newborn screening and build upon gene therapy support within the MDA Care Center Network. MDA has invested over $6 million in the Network, which includes multidisciplinary care teams across the nation. MDA’s Resource Center includes gene therapy specialists to facilitate access to these novel treatments for families.

About Muscular Dystrophy Association

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. To learn more visit mda.org and follow MDA on Instagram, Facebook, Twitter, TikTok, LinkedIn, and YouTube.