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Muscular Dystrophy Association Convenes Regulatory, Clinical and Scientific Leaders to Showcase Latest Progress in the Field of Neuromuscular Disease

Annual MDA Clinical & Scientific Conference Spotlights Accelerated Advancements Made Possible Through Increased Collaboration Between Scientific and Clinical Communities, Specifically in the Area of Gene-Targeting Therapies

NEW YORK, April 16, 2019 /PRNewswire/ -- Featuring an audience of over 1,200 attendees and a lineup of renowned speakers, the first day of the Annual Muscular Dystrophy Association (MDA) Clinical and Scientific Conference exemplified the meeting's theme of "Progress in Motion" by highlighting the achievements made in neuromuscular disease research and care at each stage of development, from scientific discovery to regulatory approval. The conference is being held April 14-17, 2019, at the Hyatt Regency Orlando.

As part of the opening ceremony, Dr. Janet Woodcock delivered a keynote presentation, which focused on the treatment pathway from discovery to delivery, recent work of the U.S. Food and Drug Administration (FDA) in the neuromuscular space, innovations in development programs and master protocol. During her speech, she highlighted the hope that has been ignited by the many therapies currently being investigated, while acknowledging that new approaches in clinical development must be established in order to advance the field.

A panel featuring experts from the FDA and fellow industry partners provided an in-depth look at the inner workings of the FDA and the agency's commitment to neuromuscular disease. Panelists, including Dr. Janet Woodcock and Dr. Peter Marks, explored topics at the intersection of research and clinical care, including the role of real-world evidence, clinical trial design and the importance of data sharing through platforms such as MDA's MOVR (NeuroMuscular Observational Research) Data Hub.

"Calibrating efforts to help speed the development and availability of life-changing innovations is of paramount importance at the FDA," said Peter Marks, MD, PhD, Center for Biologics Evaluation and Research, FDA. "That is why the opportunity to address researchers and clinicians working across neuromuscular conditions is particularly significant. This discussion helps to extend the understanding of the FDA's scope, priorities, resources and considerations and reinforces how we can effectively work together to progress advancements in treatments, as well as potential cures."

Among the various clinical and scientific discussions were two sessions devoted to research surrounding gene-targeting therapies. Experts shared insights from across the spectrum of neuromuscular diseases including Duchenne muscular dystrophy (DMD), spinal muscular dystrophy (SMA) and amyotrophic lateral sclerosis (ALS). The discussions underscored promising clinical progress in the space such as the practice of gene silencing – which has, for example, proven to be feasible for at least two ALS genes through multiple approaches that are currently being explored.

As the MDA Clinical and Scientific Conference continues, scientific and clinical sessions will delve into a broad range of topics in neuromuscular disease research including technology in healthcare, imaging and biomarkers, therapeutic approaches and the latest developments in newborn screening. For the latest on the conference, please follow the MDA's social channels on Twitter, Facebook and LinkedIn, or search the hashtag #MDAConference.

The full conference agenda is available on the MDA website at:

About MDA

MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is a transformative platform, combining MDA's Care Center Network with a state-of-the-art information management system to improve health outcomes for neuromuscular disease patients and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best-in-class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events and materials for families and health care providers. Each year, thousands of children and young adults learn vital life skills and gain independence at MDA Summer Camp and through recreational programs, at no cost to families. For more information, visit

SOURCE: Muscular Dystrophy Association