NeurologyLive® Friday 5 — January 30, 2026 Isabella Ciccone, MPH

Published Date: 03 Feb 2026

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 30, 2026.

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Latest News

Taysha Doses First Patient in Pivotal Trial for Rett Syndrome Gene Therapy TSHA-102, Aligns With FDA on BLA Plans Noah Stansfield

Specifically, the alignment relates to a separate, planned clinical trial referred to as ASPIRE.

Ocugen’s Stargardt Gene Therapy OCU410ST Provides +6-Letter Gain in BCVA-Evaluable Patients in Phase 1 Trial Noah Stansfield

With regard to safety, there were no serious adverse events (AEs) or AEs of special interest deemed related to OCU410ST reported in the study.

Higher CTE Stages Linked to Increased Dementia Risk, Postmortem Analysis Shows Louie Pasculli

A study of 614 brain donors revealed that individuals with stage III or IV CTE face the greatest risk of dementia.

New 3-Year EMBARK Data Highlights Continued Therapeutic Effect of Gene Therapy Elevidys in Duchenne Marco Meglio

New findings from the EMBARK study highlight Elevidys' significant long-term benefits in slowing Duchenne muscular dystrophy progression in young patients.

Phase 2 Study of Lenadogene Nolparvovec Gene Therapy for Leber Hereditary Optic Neuropathy: The REVISE Trial Isabella Ciccone, MPH

The REVISE trial, a phase 2 study evaluating GenSight Biologics’ lenadogene nolparvovec in patients with Leber hereditary optic neuropathy, is expected to begin in January 2026.

FDA Lifts Clinical Hold on Intellia’s Trial for Gene Editing Therapy Nex-Z in ATTRv-PN, Places Holds on REGENXBIO MPS Trials Noah Stansfield

Intellia’s trial for nex-z in ATTR-CM remains under clinical hold.

Todays Updates

AAN Publishes First-Ever Guideline for Functional Seizure Care Management Marco Meglio

The AAN's new guideline enhances diagnosis and management of functional seizures, emphasizing psychological interventions and addressing care gaps for better patient outcomes.

Navigating Autoimmune Overlap in Celiac Disease, With Daniel Mallon, MD Daniel Mallon, MD

Mallon explains the shared risks linking celiac disease and type 1 diabetes and why early screening improves outcomes.

Unraveling the Complexities of Concomitant IBD and Hepatobiliary Disease, With Natalia Nedelkopoulou, MD Natalia Nedelkopoulou, MD

Nedelkopoulou emphasizes the importance of structured and multidisciplinary transition programs for young patients with IBD and HPB disease.

FDA Accepts Oxylanthanum Carbonate NDA Resubmission for Hyperphosphatemia in CKD, Dialysis

FDA Accepts Oxylanthanum Carbonate NDA Resubmission for Hyperphosphatemia in CKD, Dialysis

6 Nephrology Headlines You Missed In January 2026

6 Nephrology Headlines You Missed In January 2026

OptIMMize-1: New Efficacy and Safety Data on Risankizumab in Pediatric Psoriasis

OptIMMize-1: New Efficacy and Safety Data on Risankizumab in Pediatric Psoriasis

Positive Topline Data Released on Roflumilast Cream in Infants with Atopic Dermatitis

Positive Topline Data Released on Roflumilast Cream in Infants with Atopic Dermatitis

Todays Updates

AAN Publishes First-Ever Guideline for Functional Seizure Care Management Marco Meglio

The AAN's new guideline enhances diagnosis and management of functional seizures, emphasizing psychological interventions and addressing care gaps for better patient outcomes.

Navigating Autoimmune Overlap in Celiac Disease, With Daniel Mallon, MD Daniel Mallon, MD

Mallon explains the shared risks linking celiac disease and type 1 diabetes and why early screening improves outcomes.

Unraveling the Complexities of Concomitant IBD and Hepatobiliary Disease, With Natalia Nedelkopoulou, MD Natalia Nedelkopoulou, MD

Nedelkopoulou emphasizes the importance of structured and multidisciplinary transition programs for young patients with IBD and HPB disease.

FDA Accepts Oxylanthanum Carbonate NDA Resubmission for Hyperphosphatemia in CKD, Dialysis

FDA Accepts Oxylanthanum Carbonate NDA Resubmission for Hyperphosphatemia in CKD, Dialysis

6 Nephrology Headlines You Missed In January 2026

6 Nephrology Headlines You Missed In January 2026

OptIMMize-1: New Efficacy and Safety Data on Risankizumab in Pediatric Psoriasis

OptIMMize-1: New Efficacy and Safety Data on Risankizumab in Pediatric Psoriasis

Positive Topline Data Released on Roflumilast Cream in Infants with Atopic Dermatitis

Positive Topline Data Released on Roflumilast Cream in Infants with Atopic Dermatitis

Latest News

Specifically, the alignment relates to a separate, planned clinical trial referred to as ASPIRE.

With regard to safety, there were no serious adverse events (AEs) or AEs of special interest deemed related to OCU410ST reported in the study.

A study of 614 brain donors revealed that individuals with stage III or IV CTE face the greatest risk of dementia.

New findings from the EMBARK study highlight Elevidys' significant long-term benefits in slowing Duchenne muscular dystrophy progression in young patients.

The REVISE trial, a phase 2 study evaluating GenSight Biologics’ lenadogene nolparvovec in patients with Leber hereditary optic neuropathy, is expected to begin in January 2026.

Intellia’s trial for nex-z in ATTR-CM remains under clinical hold.

News All Time

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

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.

NeurologyLive® Friday 5 — January 23, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 23, 2026.

NeurologyLive® Brain Games: January 25, 2025 Marco Meglio, Louie Pasculli

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 ALS Treatment pathways!

Episode 159: Key Practice Takeaways From the New AAN Functional Seizure Guidelines Marco Meglio, Benjamin Tolchin, MD, MS, FAAN

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

FDA Grants Orexin Agonist Alixorexton Breakthrough Therapy Designation for Narcolepsy Type 1 Isabella Ciccone, MPH

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.

AskBio’s Late-Onset Pompe Disease Gene Therapy AB-1009 Cleared for Phase 1/2 Trial Noah Stansfield

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

NLX-112 Shows Safety and Tolerability in Phase 2a Parkinson Trial, Supporting Further Development Louie Pasculli

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.

Migraine Medication Zavegepant Safe and Well Tolerated in Lactating Women, Phase 1 Study Shows Louie Pasculli

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.

Levacetylleucine Meets Primary and Secondary End Points in Phase 3 Ataxia-Telangiectasia Study Isabella Ciccone, MPH

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.

NeuroVoices: Sneha Mantri, MD, MS, on Addressing Depression and Anxiety in Parkinson Care Marco Meglio

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.

FDA Requests HOPE-3 Clinical Study Report for Deramiocel BLA in DMD Cardiomyopathy Isabella Ciccone, MPH

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 Opicapone Initiation in Parkinson Leads to Continued Long-Term Therapeutic Benefits Marco Meglio

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

Low-Dose Opioids Show Long-Term Dose Stability, Effectiveness in Augmented Restless Legs Syndrome Louie Pasculli

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.

Dopamine D3 Receptor Mesdopetam Maintains Antidyskinesia Effects Despite Failure to Improve Parkinson Disease ON Time Marco Meglio

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

What the EC Decision on High-Dose Spinraza Means for SMA Care Marco Meglio, Stephanie Fradette, PhD

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.

Investigational COYA 302 Shows Feasibility, Preliminary Efficacy in Small Trial of Frontotemporal Dementia Marco Meglio

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

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