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NeurologyLive® Brain Games: January 25, 2025

Phase 1/2a Findings Lend Support for BB-301 in Oculopharyngeal Muscular Dystrophy

Evaluating Durable Dysphagia Outcomes in OPMD: Insights From Early BB-301 Clinical Data

NeurologyLive® Friday 5 — January 23, 2026

FDA Grants Orexin Agonist Alixorexton Breakthrough Therapy Designation for Narcolepsy Type 1

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

AskBio noted that AB-1009 also recently received fast track and orphan drug designations from the FDA.

A phase 2a trial showed that NLX-112 was safe and well tolerated in patients with Parkinson disease, while also reducing levodopa-induced dyskinesia and motor symptoms, supporting further clinical development.

A Phase 1 study in healthy lactating women found that a single 10‑mg dose of zavegepant nasal spray was generally safe, well tolerated, and resulted in minimal infant exposure through breast milk.

IntraBio reported that levacetylleucine showed symptom improvement in the phase 3 IB1001-303 study for ataxia-telangiectasia, and it intends to proceed with regulatory submissions in multiple regions.

The chief medical officer at the Parkinson’s Foundation detailed how mood and behavioral symptoms shape quality of life in Parkinson disease and how clinicians can better identify and manage them.

A five-year analysis from the National RLS Opioid Registry found that most patients with dopamine agonist augmented restless legs syndrome maintained stable, low-dose opioid therapy with sustained symptom control.

The FDA requested the full data from the phase 3 HOPE-3 study as part of its review of the biologics license application for deramiocel, which Capricor plans to submit in February 2026.

Early initiation of opicapone significantly reduces OFF time and enhances ON time in patients with recently diagnosed Parkinson's disease, improving overall treatment outcomes.

A recent phase 2b study reveals mesdopetam's potential in reducing dyskinesia severity in Parkinson disease, despite no significant ON time improvement.

Stephanie Fradette, PharmD, head of neuromuscular development at Biogen, gave immediate reaction to the EC approval of high-dose nusinersen and its implications for evolving SMA treatment strategies.

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

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 International Stroke Conference (ISC)!

Coya Therapeutics reveals promising results for COYA 302, a potential treatment for frontotemporal dementia, showing cognitive stability and Treg enhancement.

The therapy also received orphan drug designation from the FDA.


























