The Need for Participation in Neuromuscular Disease Clinical Trials: Donald S. Wood, PhD

The president and CEO of the Muscular Dystrophy Association spoke about the need for increased participation in neuromuscular disorder clinical trials and the rapid therapeutic progress the field is seeing. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

“One of the most important things that I can communicate to the neurologists out there is that when you diagnose a patient today with a neuromuscular disease—and I don’t care what it is—the very next thing to do is to start preparing that patient for participation in a clinical trial. That’s how fast the field is moving.”

After many long years without much therapeutic progress, patients with neuromuscular diseases have seen rapid and revolutionary steps forward over the last 15 years. Although much work remains and more challenges linger, many patient populations have gained FDA approvals for the treatment of their diseases. Several diseases, such as amyotrophic lateral sclerosis (ALS) or spinal muscular atrophy (SMA), now have multiple FDA-approved treatments that have changed the landscape of care, and the pipeline of development is brimming with potential as more pharmaceutical developers are entering the neuromuscular space.

Donald S. Wood, PhD, president and CEO, Muscular Dystrophy Association (MDA), noted that this shift in therapeutic progress calls for a similar push in clinical trial participation. In a conversation with NeurologyLive® at the 2022 MDA Clinical & Scientific Conference, March 13-16, in Nashville, Tennessee, he shared his hopes that neurologists will prioritize speaking to their patients—particularly those who are newly diagnosed—about enrolling in trials.

Additionally, Wood shared his perspective on the ongoing efforts to improve the rates of genetic analysis for these diseases as well. He noted that as the therapeutic momentum speeds up, there is a parallel need to rapidly identify genetic variants and mutations that are driving many of the neuromuscular diseases. Wood also spoke to the need to gain further understanding of how different treatment approaches, and the timing of such approaches, benefit different patient populations.

For more coverage of MDA 2022, click here.