Recommendations for Clinicians Taking Care of Patients with SMA

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.

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.

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

In recognition of International Ataxia Awareness Day, observed annually on September 25, NeurologyLive® reviews 6 investigational agents in clinical development across ataxia-related disorders.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 25, 2026.