The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the challenges of familiarizing oneself with the influx of novel medications for neuromuscular diseases. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
“Asking about how to collect data in a structured fashion after a drug is approved is a big question in neuromuscular diseases. I definitely face that with ALS, especially because our recently approved therapy and others that are under review will require additional data for us to understand who would benefit the best from those therapies.”
The field of neuromuscular disease has experienced a boom in therapeutic availability over the past several years, and while this influx of new options has been welcomed, it has also presented unique challenges for physicians and patients. Particularly, as many of these therapies are genetically mediated, determining the proper patient selection and collecting long-term safety data have proven difficult.
At the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 13-16, in Nashville, Tennessee, a session was held that featured a number of expert clinicians discussing treatment selection for disorders such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy, amyotrophic lateral sclerosis (ALS), and myasthenia gravis, which have all had new FDA-approved therapies hit the market. Jinsy Andrews, MD, MSc, FAAN, who herself focuses mainly on ALS care, was among those clinicians in the session.
Andrews, who is the director of neuromuscular clinical trials at Columbia University Irving Medical Center, sat down with NeurologyLive® at MDA 2022 to offer her perspective on some of the challenges that physicians are now facing in medical decision making and treatment selection. Among those, she highlighted some of the difficulties in determining who might benefit best from these treatments, and the collection of real-world evidence and data to help inform patients and providers about the use of these therapies.
For more coverage of MDA 2022, click here.