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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]

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.

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.