Congress Passes ACT for ALS Reauthorization, Extending Research Programs Louie Pasculli

Published Date: 04 Oct 2026

The Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026 extends federal support for ALS research and expanded access to investigational therapies, with additional provisions addressing clinical trial oversight and regulatory planning.

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Phase 4 Study Shows Stable Motor Function in Adults With SMA on Risdiplam Isabella Ciccone, MPH

Real-world 12‑month results from WeSMA, a phase 4 study, showed that oral risdiplam is well tolerated in adults with SMA, with stable motor function and consistent safety findings.

Phase 4 Study Evaluates Transition From IVIG to Subcutaneous Efgartigimod in CIDP Louie Pasculli

A phase 4 study is evaluating whether adults with chronic inflammatory demyelinating polyneuropathy can transition from intravenous immunoglobulin to subcutaneous efgartigimod PH20 without first experiencing disease worsening.

Phase 4 OCTAGON Trial to Test Rapid Steroid Taper With Ravulizumab Marco Meglio

A new phase 4 trial, OCTAGON, will test a rapid, predefined corticosteroid-tapering schedule in patients with generalized myasthenia gravis treated with ravulizumab.

LGMD Agent BBP-418 Improves Ambulation and Pulmonary Function Across Subgroups in Phase 3 FORTIFY Interim Analysis Isabella Ciccone, MPH

New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

Episode 176: BBP-418 and the Changing Treatment Landscape of LGMD Marco Meglio, Katherine Mathews, MD

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Katherine Matthews, MD. [LISTEN TIME: 17 minutes]

ADHERE REAL Study to Assess Real-World Effectiveness of Efgartigimod in CIDP Isabella Ciccone, MPH

A new study will follow adults with chronic inflammatory demyelinating polyradiculoneuropathy who start subcutaneous efgartigimod in routine practice in the US and Germany.

Todays Updates

8 Pulmonology Headlines You Missed in September 2026

8 Pulmonology Headlines You Missed in September 2026

2026 Rachel Horne Prize Winner Sandra Vukusic, MD, PhD, on Advancing Women's Health in MS Isabella Ciccone, MPH, Sandra Vukusic, MD, PhD

The professor of neurology at the University of Lyon and 2026 Rachel Horne Prize Winner discussed pregnancy counseling, therapeutic inertia, and overlooked women's health issues in multiple sclerosis.

Tips for Clinicians Diagnosing Patients with Diverse Skin Types Mona Shahriari, MD, FAAD, Jennifer Soung, MD

Jennifer Soung, MD, and Mona Shahriari, MD, discuss diagnosing psoriasis in diverse skin tones and why severity scores overlook dyspigmentation.

AMETHYST: Litifilimab Sustains CLE Gains at 52 Weeks, With Victoria Werth, MD

AMETHYST: Litifilimab Sustains CLE Gains at 52 Weeks, With Victoria Werth, MD

EADV Congress 2026: Day 3 Recap—5 Headlines to Know

EADV Congress 2026: Day 3 Recap—5 Headlines to Know

Upadacitinib Tops Nemolizumab on Skin, Itch Targets in Indirect Analysis

Upadacitinib Tops Nemolizumab on Skin, Itch Targets in Indirect Analysis

Todays Updates

8 Pulmonology Headlines You Missed in September 2026

8 Pulmonology Headlines You Missed in September 2026

2026 Rachel Horne Prize Winner Sandra Vukusic, MD, PhD, on Advancing Women's Health in MS Isabella Ciccone, MPH, Sandra Vukusic, MD, PhD

The professor of neurology at the University of Lyon and 2026 Rachel Horne Prize Winner discussed pregnancy counseling, therapeutic inertia, and overlooked women's health issues in multiple sclerosis.

Tips for Clinicians Diagnosing Patients with Diverse Skin Types Mona Shahriari, MD, FAAD, Jennifer Soung, MD

Jennifer Soung, MD, and Mona Shahriari, MD, discuss diagnosing psoriasis in diverse skin tones and why severity scores overlook dyspigmentation.

AMETHYST: Litifilimab Sustains CLE Gains at 52 Weeks, With Victoria Werth, MD

AMETHYST: Litifilimab Sustains CLE Gains at 52 Weeks, With Victoria Werth, MD

EADV Congress 2026: Day 3 Recap—5 Headlines to Know

EADV Congress 2026: Day 3 Recap—5 Headlines to Know

Upadacitinib Tops Nemolizumab on Skin, Itch Targets in Indirect Analysis

Upadacitinib Tops Nemolizumab on Skin, Itch Targets in Indirect Analysis

Latest News

Real-world 12‑month results from WeSMA, a phase 4 study, showed that oral risdiplam is well tolerated in adults with SMA, with stable motor function and consistent safety findings.

A phase 4 study is evaluating whether adults with chronic inflammatory demyelinating polyneuropathy can transition from intravenous immunoglobulin to subcutaneous efgartigimod PH20 without first experiencing disease worsening.

A new phase 4 trial, OCTAGON, will test a rapid, predefined corticosteroid-tapering schedule in patients with generalized myasthenia gravis treated with ravulizumab.

New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Katherine Matthews, MD. [LISTEN TIME: 17 minutes]

A new study will follow adults with chronic inflammatory demyelinating polyradiculoneuropathy who start subcutaneous efgartigimod in routine practice in the US and Germany.

News All Time

Somnolence Risk, Short-Term Efficacy Drive VMAT2 Inhibitor Choice in Older Adults With Tardive Dyskinesia Isabella Ciccone, MPH

In a discrete choice experiment, somnolence risk and short-term efficacy were the top factors driving vesicular monoamine transporter 2 inhibitor selection for tardive dyskinesia in patients aged 55 years and older.

This Week on NeurologyLive® — September 28, 2026 Isabella Ciccone, MPH

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

Inebilizumab Shows Durable Benefit in MuSK+ Generalized Myasthenia Gravis at AANEM 2026 Marco Meglio

New phase 3 MINT trial analyses presented at the 2026 AANEM Annual Meeting showed inebilizumab produced sustained improvement in MuSK-positive gMG regardless of time since diagnosis.

SHIELD Trial Advances Combination Antiviral for Long COVID Marco Meglio

PridCor Therapeutics is moving its phase 2 SHIELD trial of a combination antiviral regimen toward enrollment, building on case series data showing durable symptom improvement in long COVID.

Gefurulimab Cuts Hospitalizations, Sustains Response in Phase 3 PREVAIL Trial of Myasthenia Gravis Marco Meglio

New PREVAIL trial analyses presented at the 2026 AANEM Annual Meeting showed gefurulimab reduced gMG-related hospitalizations and produced durable symptom and quality-of-life gains through one year.

FDA Approves Tavapadon for the Treatment of Parkinson Disease Isabella Ciccone, MPH

FDA approves once-daily oral tavapadon for Parkinson disease, a first-in-class D1/D5 partial agonist that improves motor function and ON time, with key safety warnings.

Parkinson's Disease Gets New Treatment Approved

FDA gives go-ahead to D1/D5 dopamine agonist tavapadon

Unexpected Cognitive Improvement Emerges in Hearing Aid Trial

— Treating hearing loss may help some people regain cognitive and functional capacity, data suggest

From Biomarkers to Prevention: Key Alzheimer Research Stories of 2026 Louie Pasculli

In recognition of World Alzheimer's Day, NeurologyLive reviews Alzheimer disease developments from 2026, spanning blood-based biomarkers, emerging treatments, prevention research, disease risk factors, and safety considerations surrounding anti-amyloid therapies.

NeuroVoices: Eva Mistry, MD, on Addressing Secondary Stroke Prevention Gaps in Underserved Populations Eva Mistry, MD , Isabella Ciccone, MPH

The professor of neurology at the University of Cincinnati discussed why disparities persist in secondary stroke prevention and how growing awareness of social determinants of health is helping to close the gap for underserved patients.

Simufilam Clears FDA Hold, Advances to Phase 2 Trial of Tuberous Sclerosis Complex Marco Meglio

The FDA lifted a clinical hold on simufilam, clearing Filana Therapeutics, formerly Cassava Sciences, to begin a phase 2a study in TSC-related epilepsy.

CHMP Recommends EU Approval for Ocrelizumab in Pediatric Relapsing MS Louie Pasculli

The positive opinion for intravenous ocrelizumab was supported by phase 3 OPERETTA 2 findings demonstrating noninferior relapse control and superior suppression of MRI-detected brain lesions compared with fingolimod in pediatric patients with relapsing MS.

Migraine Forecasting Model Reaches 91% Precision Using App-Based Headache Data Louie Pasculli

A migraine forecasting model achieved 91% precision using app data, but the study did not test whether next-day predictions improve care.

SKY-0515 Shows Sustained Benefit at 15 Months in Huntington Disease Marco Meglio

Final 15-month phase 1/2 data showed SKY-0515 improved cUHDRS scores and lowered mutant huntingtin protein in early-stage Huntington disease.

FDA Grants Fast Track Designation to Apitegromab for Facioscapulohumeral Muscular Dystrophy Isabella Ciccone, MPH

The FDA has granted Scholar Rock's apitegromab fast track designation for the treatment of patients with living facioscapulohumeral muscular dystrophy as the company's phase 2 FORGE study dosing begins.

Real-World Study Supports Peroneal Nerve Stimulation for Restless Legs Syndrome Symptoms Isabella Ciccone, MPH

Tonic motor activation therapy was associated with reductions in RLS severity, along with tolerable reductions in dopamine agonist dosing, in adults with medication-refractory RLS.

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