ELEVIDYS Shows Functional Benefits in Patients With DMD, Dompé Launches Phase 2 Trial of Intranasal Nerve Growth Factor in CP, NeurologyLive Roundtable: The Road Ahead for Precision Medicine in MS

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

New data from the 2026 World Muscle Society Annual Congress suggest that delandistrogenemoxeparvovec-rokl (ELEVIDYS; Sarepta Therapeutics), a gene therapy for Duchenne muscular dystrophy (DMD), was associated with better functional outcomes over 2 years in older ambulatory patients compared with matched external controls. Separate findings in children aged 2 to younger than 3 years demonstrated micro-dystrophin expression following treatment, although the analysis did not establish a functional benefit in this younger population.¹

In the older-patient analysis, investigators evaluated 25 ambulatory patients aged 8 to 12 years from the EMBARK and ENDEAVOR studies and compared their outcomes with those of 99 well-matched external controls drawn from DMD natural history datasets. The analysis used a prospectively specified matching approach intended to identify comparable patients and account for key factors that could influence disease progression.

At 2 years, patients treated with ELEVIDYS had significantly better outcomes than external controls across 3 measures of motor function. The least-squares mean difference favored treatment by 3.32 points on the North Star Ambulatory Assessment (NSAA; 95% CI, 1.24-5.41; P = .0022), 0.041 rises per second in time-to-rise-from-floor velocity (95% CI, 0.016-0.066; P = .0019), and 0.195 m/s in 10-meter walk/run velocity (95% CI, 0.046-0.345; P = .0114).¹

Dompé has enrolled the first patient in a phase 2 trial testing whether intranasal nerve growth factor (NGF) can safely be given to young children with spastic cerebral palsy (CP), a condition for which no treatments currently address the underlying brain injury. The trial, known as Galassia, is testing the company's investigational cenegermin nasal spray, the same recombinant NGF molecule already approved as an eye drop for a rare corneal disease.²

“Cerebral palsy is a devastating disease, and for patients and families there are still no proven treatments beyond supportive care,” Marcello Allegretti, chief scientific officer of Dompé, said in a statement.³ "With our emerging intranasal NGF platform, we hope to deliver a treatment that goes beyond managing symptoms and changes the course of disease, opening the door to a better future for patients and their families.”

Investigators presented the trial's design at the 2026 Child Neurology Society (CNS) Annual Meeting in Montreal, Quebec, Canada. For context, Galassia (EU-CTR 2025-522786-29-00; protocol NGF-CP-201) is an ongoing phase 2, randomized, double-blind, vehicle-controlled, 2-part crossover study enrolling approximately 60 children ages 2 to 6 with spastic CP across 12 sites in Italy, including centers in Rome, Milan, Pisa, Brescia, and Genoa.^2-4

Unpredictability is one of the defining challenges of multiple sclerosis (MS). Patients often can’t know how their disease will behave, and clinicians can’t always know which therapy will work best or when to change course. Precision medicine aims to close that gap by drawing on each patient’s clinical history, imaging, biology, and preferences, so that treatment can be built around the individual rather than the average. The field has made real progress toward that goal, but many of its most important questions remain open.

NeurologyLive®’s Roundtable series Precision Medicine in Multiple Sclerosis: Advances & Unanswered Questions, produced in collaboration with Cleveland Clinic Neurological Institute, concludes with a look at those open questions. Throughout the series, Robert Bermel, MD, director of Cleveland Clinic’s Mellen Center for Multiple Sclerosis Treatment and Research; Marisa McGinley, DO, staff neurologist at the Mellen Center; and Rebecca Cebull, CNP, certified nurse practitioner at the Mellen Center, have examined biomarkers, advanced imaging, genetics, and PIRA. In this final installment, they consider what it will take to move the field forward.

To watch this episode or the entire roundtable, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. Sarepta Therapeutics. Sarepta Therapeutics presents new data at 2026 World Muscle Society Annual Congress demonstrating safety and clinically meaningful functional benefit of ELEVIDYS (delandistrogenemoxeparvovec) in older ambulatory patients with Duchenne. Published September 30, 2026. https://www.businesswire.com/news/home/20260930354567/en/Sarepta-Therapeutics-Presents-New-Data-at-2026-World-Muscle-Society-Annual-Congress-Demonstrating-Safety-and-Clinically-Meaningful-Functional-Benefit-of-ELEVIDYS-delandistrogene-moxeparvovec-in-Older-Ambulatory-Patients-with-Duchenne.
2. Romeo D, Guzzetta A, Galli J, et al. Clinical trial in progress: Galassia, CP-201, a phase 2, randomized, double-blind, vehicle-controlled study of intranasal cenegermin in pediatric spastic cerebral palsy. Abstract 19. Presented at: 2026 Child Neurology Society Annual Meeting; October 14-17, 2026; Montreal, Quebec, Canada.
3. Dompé announces first patient enrolled in new study of intranasal NGF in cerebral palsy. News release. Published March 26, 2026. Accessed October 13, 2026. https://www.businesswire.com/news/home/20260326265178/en/Domp-Announces-First-Patient-Enrolled-in-New-Study-of-Intranasal-NGF-in-Cerebral-Palsy
4. EU Clinical Trials Information System. Intranasal cenegermin in pediatric participants with spastic cerebral palsy. EU-CTR 2025-522786-29-00. Accessed October 13, 2026. https://ctis.eu/trial/2025-522786-29-00