Final Results Reported for EPISOD1 Trial of SOD1-ALS Gene Therapy AMT-162 Marco Meglio

Published Date: 06 Nov 2026

uniQure presented final safety and biomarker results from the discontinued EPISOD1 trial of AMT-162, an investigational gene therapy for SOD1-associated ALS.

Read Full News

Latest News

On Beyond Seizures: Unmet Needs in Pediatric Epilepsy Clinics Susan Duberstein, MD

Pediatric epilepsy care goes beyond seizures, addressing mental health, learning needs, coordinated referrals, equitable access, and smoother transition to adult services.

FDA Grants Priority Review to Huntington Disease Gene Therapy BLA Marco Meglio

The FDA accepted uniQure's biologics license application for ifezuntirgene inilparvovec and granted it Priority Review, setting a target action date of May 1, 2027.

NeuroVoices: Cindy Zadikoff, MD, MSc, on the FDA Approval of Tavapadon for Parkinson Disease Isabella Ciccone, MPH, Cindy Zadikoff, MD, MSc

The executive medical director at AbbVie discussed the FDA approval of tavapadon, the first D1/D5 dopamine agonist for Parkinson disease, including the supporting TEMPO trial data and safety considerations.

Brezosiran Demonstrates Mutant Huntingtin Reduction in Phase 1b Huntington Disease Study Louie Pasculli

Alnylam Pharmaceuticals reported initial Phase 1b findings showing brezosiran reduced cerebrospinal fluid mutant huntingtin levels by a mean of 60% to 64% at 3 months, with plans for a pivotal study in 2027.

AL-S Pharma Details Planned Phase 3 Trial Design for AP-101 in ALS Marco Meglio

AL-S Pharma outlined the design of AP101-03, a planned phase 3 trial of AP-101 in sporadic and SOD1-associated ALS, at the 2026 NEALS Annual Meeting.

Alzheimer Agent Sabirnetug Misses Primary Endpoint, Acumen Halts Development Marco Meglio

Sabirnetug failed to meet its primary endpoint in Acumen Pharmaceuticals' phase 2 ALTITUDE-AD trial for early Alzheimer disease, prompting the company to end development.

Todays Updates

Conference Preview: 2026 GOLD International COPD Conference Tim Smith

HCPLive previews GOLD 2026 in Philadelphia, November 5-6, with sessions on the 2027 GOLD Report, mucus plugging, and exacerbations, plus expert interviews.

7 Pulmonology Headlines You Missed in October

7 Pulmonology Headlines You Missed in October

Nasal mRNA ETH47 Lowers Rhinovirus Load in Asthma Challenge Study

Nasal mRNA ETH47 Lowers Rhinovirus Load in Asthma Challenge Study

Q&A: Why 70% of COPD Goes Undiagnosed, With Alvar Agustí, MD

Q&A: Why 70% of COPD Goes Undiagnosed, With Alvar Agustí, MD

Mucus Plugs in COPD: Prevalence, Exacerbations, Mortality Risk

Mucus Plugs in COPD: Prevalence, Exacerbations, Mortality Risk

Analyzing Inebilizumab’s Durable Benefit in MINT Open-Label Extension Marco Meglio

Richard Nowak, MD, director of the Yale Myasthenia Gravis Clinic and global principal investigator of the MINT trial, discusses open-label extension findings for inebilizumab, a CD19-directed B cell depleting therapy, in generalized myasthenia gravis.

IJMSC Webinar Series Guides MS Clinicians From Idea to Publication Matt Hoffman

IJMSC editor-in-chief, François Bethoux, MD, and colleagues cover idea generation, poster conversion, literature reviews, and peer review in a 4-part webinar series for MS clinicians.

Todays Updates

Conference Preview: 2026 GOLD International COPD Conference Tim Smith

HCPLive previews GOLD 2026 in Philadelphia, November 5-6, with sessions on the 2027 GOLD Report, mucus plugging, and exacerbations, plus expert interviews.

7 Pulmonology Headlines You Missed in October

7 Pulmonology Headlines You Missed in October

Nasal mRNA ETH47 Lowers Rhinovirus Load in Asthma Challenge Study

Nasal mRNA ETH47 Lowers Rhinovirus Load in Asthma Challenge Study

Q&A: Why 70% of COPD Goes Undiagnosed, With Alvar Agustí, MD

Q&A: Why 70% of COPD Goes Undiagnosed, With Alvar Agustí, MD

Mucus Plugs in COPD: Prevalence, Exacerbations, Mortality Risk

Mucus Plugs in COPD: Prevalence, Exacerbations, Mortality Risk

Analyzing Inebilizumab’s Durable Benefit in MINT Open-Label Extension Marco Meglio

Richard Nowak, MD, director of the Yale Myasthenia Gravis Clinic and global principal investigator of the MINT trial, discusses open-label extension findings for inebilizumab, a CD19-directed B cell depleting therapy, in generalized myasthenia gravis.

IJMSC Webinar Series Guides MS Clinicians From Idea to Publication Matt Hoffman

IJMSC editor-in-chief, François Bethoux, MD, and colleagues cover idea generation, poster conversion, literature reviews, and peer review in a 4-part webinar series for MS clinicians.

Latest News

Pediatric epilepsy care goes beyond seizures, addressing mental health, learning needs, coordinated referrals, equitable access, and smoother transition to adult services.

The FDA accepted uniQure's biologics license application for ifezuntirgene inilparvovec and granted it Priority Review, setting a target action date of May 1, 2027.

The executive medical director at AbbVie discussed the FDA approval of tavapadon, the first D1/D5 dopamine agonist for Parkinson disease, including the supporting TEMPO trial data and safety considerations.

Alnylam Pharmaceuticals reported initial Phase 1b findings showing brezosiran reduced cerebrospinal fluid mutant huntingtin levels by a mean of 60% to 64% at 3 months, with plans for a pivotal study in 2027.

AL-S Pharma outlined the design of AP101-03, a planned phase 3 trial of AP-101 in sporadic and SOD1-associated ALS, at the 2026 NEALS Annual Meeting.

Sabirnetug failed to meet its primary endpoint in Acumen Pharmaceuticals' phase 2 ALTITUDE-AD trial for early Alzheimer disease, prompting the company to end development.

News All Time

World Stroke Day 2026: Closing the Gap Between Stroke Onset and the Emergency Call Number Isabella Ciccone, MPH, Brian Snelling, MD

Brian Snelling, MD, director of the stroke program at Marcus Neuroscience Institute discussed delays in calling 911 after stroke onset, gaps in awareness of atypical symptoms, and how to reach underserved populations.

World Stroke Day: Preventing Perioperative Stroke With Predictive Tools Marco Meglio, Shadi Yaghi, MD

Shadi Yaghi, MD, FAHA, Chair of Neurology at Hackensack Meridian Neuroscience Institute at Jersey Shore University Medical Center, discusses a new calculator that predicts stroke risk within 30 days of a procedure.

Incorporating the Updated AAN/AHS Migraine Prevention Guideline Into Practice Isabella Ciccone, MPH, Ann Pakalnis, MD

The clinical professor of neurology and pediatrics at The Ohio State University Wexner Medical Center discussed payer step therapy, earlier preventive treatment, and questions the updated guideline leaves open.

Collective Advocacy, Shared Priorities, and What’s Ahead at MDA 2027 Marco Meglio, Paul Melmeyer, MPP

Paul Melmeyer, MPP, Executive Vice President of Public Policy and Advocacy at the Muscular Dystrophy Association, discusses the Neuromuscular Advocacy Collaborative, its growing year-round presence, and what the group hopes to accomplish heading into the 2027 MDA Clinical and Scientific Conference.

Joanna Wardlaw, MD, on Biomarkers, Repurposed Drugs, and the Future of Small Vessel Disease Treatment Marco Meglio

Joanna Wardlaw, MD, Professor of Applied Neuroimaging at the University of Edinburgh, discusses the evolving understanding of cerebral small vessel disease, emerging therapeutic approaches, and the makings behind a successful trial.

Brain Chat: Migraine: More Than Just a Headache Mitzi Joi Williams, MD

Mitzi Joi Williams, MD, is joined by Temitope Adeola, MD, and Chantale Branson, MD, to discuss migraine as a complex neurological condition, including recognizing symptoms and triggers, navigating acute and preventive treatment options, and incorporating lifestyle modifications to improve migraine management.

NeuroVoices: Bruce Ovbiagele, MD, MSc, MAS, MBA, on the Future of Stroke Publishing Marco Meglio, Bruce Ovbiagele, MD, MSc, MAS, MBA

The Editor-in-Chief of the Journal of the American Heart Association discussed what journal editors look for in stroke research submissions, how AI and open access are reshaping publishing, and the equity gaps that still exist for researchers in lower-income countries.

Phase 2 ACUITY Analysis Shows Privosegtor Associated With Smaller Early NfL and GFAP Increases in Acute Optic Neuritis Isabella Ciccone, MPH

New biomarker data showed smaller post-onset increases in plasma neurofilament light chain and glial fibrillary acidic protein with privosegtor added to steroids in patients with acute optic neuritis.

The Expanding Role of Blood-Based Biomarkers in MS Care Louie Pasculli, Terrie Livingston, PharmD

Terry Livingston, PharmD, vice president of medical affairs at Octave Bioscience, discussed the evolving role of blood-based biomarkers in multiple sclerosis care.

CORE-VNS Study Shows Sustained Seizure Reduction With VNS Therapy at 36 Months Louie Pasculli

The 36-month CORE-VNS study findings showed sustained seizure reduction, improved quality of life, and reduced seizure-related hospitalizations among people with drug-resistant epilepsy receiving vagus nerve stimulation therapy.

Calcium Channel Blockers Linked to Lower Blood Pressure Variability Marco Meglio

A target trial emulation pooling ACCORD-BP and SPRINT data found calcium channel blocker regimens reduced blood pressure variability without changing cardiovascular outcomes.

This Week on NeurologyLive® — October 26, 2026 Isabella Ciccone, MPH

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

CAR-T Agent Zola-cel Shows No New Multiple Sclerosis Disease Activity in Phase 1 Study Louie Pasculli

Updated data from Breakfree-2 study showed that treatment with a single zola-cel infusion achieved NEDA-3 in highly active relapsing multiple sclerosis and led to mostly stable disability in progressive multiple sclerosis.

Remibrutinib Lowers Relapse Rate vs Teriflunomide in Relapsing MS Matt Hoffman, Louie Pasculli

Phase 3 data show the oral BTK inhibitor reduced annualized relapse rate by 35.2% and 31.2% vs teriflunomide, with no liver safety signal or Hy's Law cases.

Houston Forum Highlights Awareness Gaps in NF1 Plexiform Neurofibroma Care Marco Meglio

At a NeurologyLive and Targeted Oncology Clinical Forum in Houston, Kevin J. Bielamowicz, MD, of Arkansas Children’s Hospital, discussed how incidental diagnosis and limited awareness of systemic therapy continue to delay care for patients with NF1-associated plexiform neurofibromas.

Tenecteplase Benefit Before Thrombectomy Varies by Stroke Cause, Study Shows Marco Meglio

A secondary analysis of the BRIDGE-TNK trial found tenecteplase before thrombectomy improved functional outcomes in cardioembolic but not atherosclerotic stroke.

Recommended News For You

Anticoagulation for 12 months has been found to improve outcomes in cancer patients with minor blood clots.

CD39 Blockade Shows Potential in Early-Stage Lung Cancer

What Caused a Man to Develop Friable Bleeding Nodules on His Arms and Hips?

Error on the MGUS-Autoimmune Disease Association.

Global inequalities found in cancer research funding

AI Unlocks Benefits of Prostate Cancer Treatment.

Researchers publish action plan to address appendiceal cancer enigmas

Recommended Articles For You

Building Physical Resilience in Chronic Blood Disorders 

Spatially Guided Tumor Margin Navigation Technologies: A Comprehensive Medical Review 

Clonal Hematopoiesis and Age-Related Genome Evolution: Implications for Hematologic and Cardiovascular Health 

Early Recognition of Immune-Related Adverse Events During Cancer Therapy 

Whats more on Hidoc Dr.

Medical Updates
KOL Videos
Surveys
Events

Daily news to keep you up to date

Gain valuable insights with our in-depth KOL Video discussions

Participate and win cash vouchers

Get updates on the latest events happening around the world
Featured News
Featured Articles
Featured Events
Featured KOL Videos

1.

Addition of regional nodal irradiation does not decrease rates of invasive breast cancer recurrence, study finds

2.

Products for Intimate Care and Cancer; Boost for Colorectal Cancer Stool Tests; Ultrasonic Biopsy?

3.

Admissions, medical colleges, fees, and eligibility requirements information for the DNB Post Diploma in Radiation Oncology.

4.

Study: Rapamycin slows the progression of cancer by reducing aging and concentrating on precancerous cells.

5.

How the metastasis of cancer is driven by cellular plasticity.

1.

Unlocking the Potential of Sarclisa: A New Hope for Cancer Treatment

2.

Cancer Care and Geographic Inequality: Bridging the Gap in Oncology Outcomes

3.

Unlocking the Mystery of Leukemia: Exploring New Treatments and Hope for Patients

4.

Pathophysiology of Tumor Cell Extracellular Matrix Remodeling

5.

Tremelimumab: A Potential Breakthrough in Cancer Treatment

1.

International Conference on Innovations in Critical Care for Oncology and Cardiology

2.

International Conference on Oncology, Cardiology and Critical Care Policy

3.

International Conference on Oncology, Cancer Prevention and Public Health

4.

International Conference on Cancer Nursing and Rehabilitation Strategies

5.

International Conference on Best Practices in Oncology, Cardiology and Critical Care

1.

How Nutrition Can Support Patients During Cancer Care

2.

Untangling The Best Treatment Approaches For ALK Positive Lung Cancer - Part III

3.

Expert Group meeting with the management of EGFR mutation positive NSCLC - Part III

4.

Breast Cancer Awareness and Early Detection

5.

Iron Deficiency Anemia: Ferric Maltol As a New Treatment Option

Fastest Growing Medical Platform for Doctors

Address :
Hidoc Dr. Inc. | Delaware C Corp | 1309 Coffeen Ave. Suite 1200, Sheridan WY, 82801

Phone :
+1-415-463-3094

Email :
anishagadia@hidoc.co

Quick Links

Drug DatabaseDrug InteractionDiseases Database
KOL VideosKnowledge HubQuizzes
Medical UpdatesEventsAbout Us

© Copyright 2026 Hidoc Dr. Inc.

Terms & Conditions - LLP | Inc. | Privacy Policy - LLP | Inc. | Account Deactivation
Home
Profile
Login
bot