A mouse study reveals a connection between dementia risk and cold frequency.

Published Date: 05 Apr 2023

Being ill frequently may slow down brain aging and raise the risk of dementia and other forms of cognitive decline.

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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.

Todays Updates

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.

Todays Updates

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.

Latest News

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.

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.

News All Time

Newborn Screening for Duchenne: Clinical and Policy Implications Marco Meglio

Paul Melmeyer, MPP, executive vice president, public policy and advocacy, Muscular Dystrophy Association, explains how adding Duchenne muscular dystrophy to the Recommended Unified Screening Panel reshapes early diagnosis, treatment timing, and family counseling.

Phase 3 VALOR Data Further Support Tofersen’s Impact on ALS Disease Progression, Survival Marco Meglio

Published phase 3 data in JAMA Neurology highlighted tofersen's potential to slow ALS progression and improve survival, marking a breakthrough in SOD1-ALS treatment.

Exploring Depressive Symptoms in Low-IQ Youth with Refractory Epilepsy Janelle L Wagner, PhD, Shannon Brothers, PhD, Cortney Wolfe-Christensen, PhD, Louie Pasculli

A trio of clinician authors provided insights on a recently published paper assessing youths with refractory epilepsy and ways to combat depressive symptoms.

FDA Approves Subcutaneous Copper Histidinate as First Treatment for Pediatric Menkes Disease Isabella Ciccone, MPH

The FDA approved Sentynl Therapeutics’ copper histidinate for the treatment of pediatric Menkes disease, a rare genetic neurodegenerative disorder, following an earlier complete response letter.

Rituximab Displays Long-Term Effectiveness for Relapsing MS Louie Pasculli

Annette Lenger-Gould, MD, PhD, lead for clinical and translational neuroscience at Kaiser Permanente, discusses findings and significance from a recently published trial assessing rituximab in patients with relapsing multiple sclerosis.

NeuroVoices: Nicholas Ashton, PhD, on Advances and Challenges in Alzheimer Disease Biomarkers Nicholas Ashton, PhD, Isabella Ciccone, MPH

The senior director of the Fluid Biomarker Program at Banner Health highlighted the evolving role of blood and fluid biomarkers in Alzheimer disease research and clinical practice.

High-Dose Nusinersen Gains European Commission Approval for Spinal Muscular Atrophy Louie Pasculli

The European Commission approved Biogen’s high-dose nusinersen regimen for 5q SMA, supported by DEVOTE trial data showing significantly improved motor function with a safety profile consistent with the standard-dose regimen.

Patients Treated With REGENXBIO’s DMD Gene Therapy RGX-202 Exceed Expected Disease Trajectory on NSAA Noah Stansfield

In the middle of 2026, the company is planning to submit a BLA for RGX-202 via an accelerated approval pathway.

Stereotactic Radiosurgery: Advancing Precision in Treating Neurological Disorders Lilyana Angelov, MD

The director of Cleveland Clinic's Gamme Knife Center and Head Section of Spine Tumors writes about how stereotactic radiosurgery has transformed modern neurology and neurosurgery

sBLA Acceptance Positions Efgartigimod as Potential First Therapy for Seronegative Myasthenia Gravis Marco Meglio

The FDA is expected to have a decision on efgartigimod as potentially the first approved therapy for seronegative myasthenia gravis by May 10, 2026.

Phase 3 Data Readout of EMPEROR Study to Come Earlier, With FDA Submission Expected to Follow Marco Meglio

Those from the clinical community may expect to hear data from the registrational trial assessing zorevunersen in Dravet syndrome by mid-2027.

Secondary Analysis Finds Oveporexton Improves Cognitive Function in Adults With Narcolepsy Type 1 Louie Pasculli

Treatment with the orexin-receptor 2 agonist oveporexton was associated with improvements in attention, memory, and executive function in adults with narcolepsy type 1.

This Week on NeurologyLive® — January 12, 2026 Isabella Ciccone, MPH

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

Annexon Submits Marketing Authorization Application for Tanruprubart to Treat Guillain-Barré Syndrome Louie Pasculli

Annexon submitted an EMA marketing application for tanruprubart, a first-in-class C1q monoclonal antibody that showed faster and more complete recovery in Guillain-Barré syndrome patients across randomized trials.

Neuroprotective Agent Privosegtor Gains FDA Breakthrough Designation for Optic Neuritis Marco Meglio

Oculis advances Privosegtor, a potential first neuroprotective therapy for optic neuritis, following FDA breakthrough designation and promising trial results.

Published Phase 2 Data Highlight Potential of NNZ-2591 in Phelan-McDermid Syndrome Marco Meglio

A phase 2 trial shows NNZ-2591 improves symptoms in children with Phelan-McDermid syndrome, paving the way for a phase 3 study.

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