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LGMD Agent BBP-418 Improves Ambulation and Pulmonary Function Across Subgroups in Phase 3 FORTIFY Interim Analysis

Phase 4 Study Shows Stable Motor Function in Adults With SMA on Risdiplam

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In a pivotal phase 3 study, once-daily tasimelteon 20 mg shifted sleep onset 37.1 minutes earlier than placebo in adults with delayed sleep-wake phase disorder.

Findings from the phase 1/2 RESET-MG trial, presented at AANEM 2026, showed that 10 of 13 patients with refractory generalized myasthenia gravis had clinically meaningful MG-ADL improvements after a single infusion of rese-cel.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Katherine Matthews, MD. [LISTEN TIME: 17 minutes]

The FDA accepted fenebrutinib's new drug application under priority review for relapsing and primary progressive MS, based on 3 phase 3 trials comparing the BTK inhibitor with teriflunomide and ocrelizumab.

A new study will follow adults with chronic inflammatory demyelinating polyradiculoneuropathy who start subcutaneous efgartigimod in routine practice in the US and Germany.

The chairman of the Neurology Service Line at Baptist Health in Kentucky discussed PREVIEW trial findings for gefelorilimab in generalized myasthenia gravis, including efficacy, safety, and what the subcutaneous C5 inhibitor could offer patients if approved.

Phase 3 trials EMNERGIZE and EMVIGORATE will evaluate the efficacy and safety of intravenous empasiprubart in adults with chronic inflammatory demyelinating polyradiculoneuropathy.

EpilepsyGTx and Viralgen have entered a manufacturing collaboration to support development of EPY201, an investigational AAV9-based gene therapy for focal refractory epilepsy planned to enter first-in-human testing in 2027.

The ongoing phase 3 study is enrolling approximately 180 adults across more than 13 countries, with topline results expected in mid-2027.

New data from the 2026 AANEM Annual Meeting showed zilucoplan delivered via autoinjector was bioequivalent to the pre-filled syringe and was well tolerated when self-administered by patients with generalized myasthenia gravis.

The phase 3 HARMONIA trial will evaluate whether a muscle- and CNS-targeted antisense conjugate improves function across multiple systems in patients with myotonic dystrophy type 1.

New phase 3 MINT trial analyses presented at the 2026 AANEM Annual Meeting showed inebilizumab produced sustained improvement in MuSK-positive gMG regardless of time since diagnosis.

PridCor Therapeutics is moving its phase 2 SHIELD trial of a combination antiviral regimen toward enrollment, building on case series data showing durable symptom improvement in long COVID.

New PREVAIL trial analyses presented at the 2026 AANEM Annual Meeting showed gefurulimab reduced gMG-related hospitalizations and produced durable symptom and quality-of-life gains through one year.

FDA approves once-daily oral tavapadon for Parkinson disease, a first-in-class D1/D5 partial agonist that improves motor function and ON time, with key safety warnings.


























