Access to Health Care and to Approved Therapies is Essential for MDA Families

A letter to the neuromuscular disease community from R. Rodney Howell, M.D., Chairman, MDA Board of Directors

There is more promise and progress than ever before in the quest to find treatments and cures for muscular dystrophy, spinal muscular atrophy (SMA) and the related diseases MDA is focused on. The drug development pipeline for neuromuscular diseases is expanding at the fastest rate in history. In less than a year, three therapies have been approved by the U.S. Food and Drug Administration (FDA) to treat Duchenne muscular dystrophy and SMA, capping off decades of research and more than $1 billion in research funding from MDA. However, individuals living with the life-threatening diseases that these drugs treat are facing a new challenge — gaining access to the approved therapies.

Today, some individuals and families are unable to access FDA approved therapies, which can significantly impact the health and life of the person denied access. Not having access to appropriate therapies is unacceptable. A treatment cannot change the lives of families and individuals if they do not have access to it.

MDA’s mission to fight every day to free individuals — and the families who love them — from the harmful effects of neuromuscular diseases aren’t just idle words. They’re the focus of everything we do, which is why MDA is committed to:

  • Advocating on behalf of families about the importance of access to approved therapy options for neuromuscular diseases;
  • Funding biomedical research and advocating for increased federal funding for scientific research, innovation and drug development;
  • Supporting clinical care for individuals living with muscular dystrophy, ALS, spinal muscular atrophy (SMA) and related diseases at our more than 150 MDA Care Centers across the country;
  • Advocating for comprehensive and meaningful health insurance coverage at the national level as part of a coalition of 10 national patient advocacy organizations that have established principles of health care coverage.

As many decades of investment in research and therapy development are yielding the treatments we have long worked toward as a community, MDA remains committed to putting the individuals and families we serve first. This means continuing to advocate about the importance of access to approved treatments.

If you have any questions, contact MDA’s Resource Center at 800-572-1717 or