FDA Approves First Drug for Rare Neurodevelopmental Disorder Rett Syndrome.

Published Date: 27 Apr 2023

In the phase 3 LAVENDER study assessing the safety and effectiveness of the medication, treatment with trofinetide (DAYBUE) showed statistically significant improvement.

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

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

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

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

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

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Todays Updates

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NeurologyLive® Friday 5 — October 2, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 2, 2026.

Todays Updates

Trappsol Cyclo Misses Primary End Point in Phase 3 Trial for Niemann-Pick Disease Type C Isabella Ciccone, MPH

Findings from the phase 3 TransportNPC study showed that Trappsol Cyclo missed the primary end point in Niemann-Pick type C, but subgroup and survival data support planned 2026 NDA.

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 2, 2026.

Latest News

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.

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.

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.

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Evaluating Zeleciment Basivarsen in Myotonic Dystrophy Type 1: The Phase 3 HARMONIA Trial Isabella Ciccone, MPH

The phase 3 HARMONIA trial will evaluate whether a muscle- and CNS-targeted antisense conjugate improves function across multiple systems in patients with myotonic dystrophy type 1.

NeuroVoices: James Winkley, MD, on Analyzing Phase 3 PREVAIL Trial of Gefurulimab Marco Meglio

The chairman of the Neurology Service Line at Baptist Health in Kentucky discussed PREVIEW trial findings for gefelorilimab in generalized myasthenia gravis, including efficacy, safety, and what the subcutaneous C5 inhibitor could offer patients if approved.

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

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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

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.

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.

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.

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.

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