Statement from Kristin Stephenson, Vice President Policy & Advocacy Muscular Dystrophy Association:The Muscular Dystrophy Association (MDA) is committed to bringing safe and effective treatments and cures to children and adults living with life-threatening neuromuscular diseases as quickly as possible. To this end, MDA has invested more than $1 billion in research grants that fund groundbreaking science to accelerate the development of potential therapies and cures. With that focus in mind, MDA hosts annual conferences that serve as global collaborative platforms where researchers and clinicians share innovative approaches to treating and eradicating these devastating diseases. We are in an exciting time—today there are potential therapies in the pipeline that were inconceivable just a few years ago, and more are expected during the next five years than in the past five decades. We are seeing the development of novel therapeutics and the repurposing and reformulation of existing drugs never before contemplated. As these treatments enter the regulatory review phase, we are hopeful that the outcome will be the speedy delivery of therapies from the lab to families’ living rooms.
As MDA’s long standing investment in medical research translates into possible therapies, we will continue to work with families, health care professionals, scientists, policy makers to ensure that the Food and Drug Administration (FDA) has the support it needs to conduct thorough and speedy regulatory review of new drug applications. MDA is proud to be a continued leader in scientific discovery and drug development, and we remain dedicated to bringing urgently needed treatments and cures to our community.
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.