What's Next in Ataxia: Pipeline Overview of Investigational Therapeutics Isabella Ciccone, MPH

Published Date: 29 Sep 2026

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

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Todays Updates

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Todays Updates

Verekitug Reduces Asthma Exacerbations Up to 87%, With Michael Wechsler, MD Michael Wechsler, MD, MSc

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Exploratory extracellular vesicle biomarkers suggested KP405, a brain-penetrant GLP-1/GIP agonist, engaged insulin signaling and neuroinflammation pathways in a small phase 1 study.

12-Month Gluten-Free Diet Linked to Cognitive, Quality-of-Life Improvements in Parkinson Disease Louie Pasculli

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From Cord Blood to Cannabinoids: 7 Agents to Watch in Cerebral Palsy Marco Meglio

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Benjamin Walter, MD, MBA, discusses how advances in biomarker testing and genetic characterization are informing Parkinson disease care, alongside developments in adaptive deep brain stimulation and efforts to identify disease earlier.

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Latest News

Full results from the phase 2b LUMA showed that investigational BIIB122/DNL151 did not slow confirmed clinical worsening in early-stage Parkinson's disease compared with placebo.

New analyses from the D1AMOND clinical program found that 69.8% of participants achieved a clinically meaningful reduction in tic severity within 8 weeks of ecopipam treatment, while an interim 18-month analysis showed sustained improvement without new safety signals.

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

PROPEL will test imeroprubart, an investigational FcRn blocker, against placebo in adults with mild to severe generalized myasthenia gravis.

Early phase 1 findings showed that the PROTAC LRRK2 degrader ARV-102 was associated with improvements in saccadic hypometria and changes in CSF markers of endolysosomal function, neuroinflammation, synaptic integrity, and axonal guidance in participants with Parkinson disease.

A new Bayesian network meta-analysis found nipocalimab's serious adverse event rates compared favorably with other gMG therapies, while overall adverse event rates were comparable.

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Episode 176: BBP-418 and the Changing Treatment Landscape of LGMD Marco Meglio, Katherine Mathews, MD

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

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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.
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PETUNIA Study to Track Nipocalimab Use in Pregnancy Marco Meglio

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Single Infusion of Rese-Cel Yields Meaningful Responses Off Immunomodulators in Phase 1/2 RESET-MG Trial Isabella Ciccone, MPH

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Evaluating Zeleciment Basivarsen in Myotonic Dystrophy Type 1: The Phase 3 HARMONIA Trial Isabella Ciccone, MPH

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.

Zilucoplan Autoinjector Matches Bioequivalence of Pre-Filled Syringe in Myasthenia Gravis Marco Meglio

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.
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EpilepsyGTx, Viralgen Partner on AAV Gene Therapy EPY201 Ahead of Planned First-in-Human Trial Louie Pasculli

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.
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Somnolence Risk, Short-Term Efficacy Drive VMAT2 Inhibitor Choice in Older Adults With Tardive Dyskinesia Isabella Ciccone, MPH

In a discrete choice experiment, somnolence risk and short-term efficacy were the top factors driving vesicular monoamine transporter 2 inhibitor selection for tardive dyskinesia in patients aged 55 years and older.

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FDA Approves Tavapadon for the Treatment of Parkinson Disease Isabella Ciccone, MPH

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