FDA Expands Leucovorin Indication, Key Phase 3 Studies Revealed at MDA 2026, Crowley on the Future of Neuromuscular Medicine

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Welcome to the Neurology News Network. My name is Louie Pasculli. Here’s a look at this week’s top stories in neurology.

Beginning with FDA news, the agency has approved expanded use of leucovorin calcium tablets for the treatment of adult and pediatric patients with cerebral folate transport deficiency associated with confirmed variants in the folate receptor 1 (FOLR1) gene. The regulatory decision marks the first FDA-approved therapy specifically indicated for this rare genetic neurological condition. According to the agency, the approval was supported by a systematic review of published literature that included case reports with patient-level data as well as mechanistic evidence supporting the biological rationale for leucovorin therapy. Marty Makary, MD, MPH, commissioner of the FDA, said this in a statement “Today’s approval represents a significant milestone for patients living with cerebral folate transport deficiency due to the FOLR1 variant, a rare genetic condition that has had no FDA-approved treatment options until today. This action may benefit some individuals with FOLR1-related cerebral folate transport deficiency who have developmental delays with autistic features.”

In other news, the Muscular Dystrophy Association held its annual conference in Orlando, Florida this past week, and Neurology Live was on the floor covering the action. During the conference, researchers recently presented the study design of 2 linked phase 3 trials, STELLAR-1 and STELLAR-2, which will assess salanersen (Biogen), an intrathecally administered antisense oligonucleotide, in presymptomatic infants with spinal muscular atrophy (SMA). The studies will offer insights on outcomes in infants receiving presymptomatic treatment with salanersen monotherapy, onasemnogene abeparvovec (Zolgensma; Novartis) monotherapy, or onasemnogene abeparvovec followed by salanersen. Lead author Thomas Crawford, MD, co-director of the MDA Clinic and professor of neurology at Johns Hopkins Medicine, presented the design of the studies at the Conference. Crawford and colleagues noted that salanersen shares a similar mechanism with the SMA approved therapy nusinersen (Spinraza; Biogen) but incorporates novel backbone chemistry intended to achieve higher potency, with the potential to maximize clinical outcomes through once-yearly dosing.

Wrapping up with more MDA news, John F. Crowley, president and chief executive officer of the Biotechnology Innovation Organization, delivered a keynote titled “Driving the Future of Innovation, Policy, and Patient Impact.” at the 2026 MDA conference. In the address, Crowley reflected on the evolution of the neuromuscular research ecosystem and emphasized the growing role of biotechnology startups, patient advocacy, and emerging technologies in shaping the future of rare disease care. Following his keynote, Crowley spoke with NeurologyLive®about the progress made in neuromuscular medicine, the collaborative ecosystem required to translate scientific discoveries into approved therapies, and the policy and access challenges that remain as the field enters what he described as a “golden age of medicine.”

To read the full interview and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to stay up to date on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.