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Ocugen’s Stargardt Gene Therapy OCU410ST Provides +6-Letter Gain in BCVA-Evaluable Patients in Phase 1 Trial

Higher CTE Stages Linked to Increased Dementia Risk, Postmortem Analysis Shows

New 3-Year EMBARK Data Highlights Continued Therapeutic Effect of Gene Therapy Elevidys in Duchenne

FDA Lifts Clinical Hold on Intellia’s Trial for Gene Editing Therapy Nex-Z in ATTRv-PN, Places Holds on REGENXBIO MPS Trials

Phase 2 Study of Lenadogene Nolparvovec Gene Therapy for Leber Hereditary Optic Neuropathy: The REVISE Trial

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The neurologist in the Department of Neurology at Saratoga Hospital Medical Group shared what excites her most about the future of neurology, specifically for movement disorders, in 2026.

Trial data showed that treatment with lamotrigine reduced myotonia severity in adults with genetically confirmed non-dystrophic myotonias, with sustained benefit and an acceptable safety profile.

The FDA raised no objections to Lexicon Pharmaceuticals’ pilavapadin for a phase 3 program, which will include 2-arm registrational studies, in patients with diabetic peripheral neuropathic pain.

Head-to-head PET imaging data indicated that valbenazine achieves significantly higher VMAT2 target occupancy than deutetrabenazine XR at therapeutic doses, with both treatments remaining well tolerated.

A recent study linked maternal diabetes to a doubled risk of multiple sclerosis in offspring, highlighting prenatal factors in MS susceptibility.

If approved, lecanemab-irmb would provide an at-home subcutaneous injection option for both treatment initiation and maintenance dosing for patients with early-stage Alzheimer disease.

Inside MS: A Clinician's Compass, a podcast hosted by neuroimmunologist Lindsay Ross, MD, brings you an exclusive interview with Edith Graham, MD. [LISTEN TIME: 40 minutes]

Here's some of what is coming soon to NeurologyLive® this week.

In a recently published study, integrating prehospital GFAP measurements with established stroke scales significantly improved the accuracy of large vessel occlusion detection in patients with acute stroke.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on the ALS Treatment pathways!

Benitec Biopharma reveals promising results for BB-301, a gene therapy showing significant improvements in swallowing function for OPMD patients.

Jerel Banks, MD, PhD, chief executive officer of Benitec Biopharma, reviews early phase 1b/2a clinical signals, mechanism, and next steps for BB-301 in oculopharyngeal muscular dystrophy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 23, 2026.

Alixorexton, an oral investigational selective orexin 2 receptor agonist, is currently in development for the treatment of narcolepsy type 1 and type 2, and idiopathic hypersomnia.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Benjamin Tolchin, MD, MS, FAAN. [LISTEN TIME: 23 minutes]





























