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

Published Date: 03 Oct 2026

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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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]

FDA Accepts Fenebrutinib NDA Under Priority Review for Relapsing and Primary Progressive MS Isabella Ciccone, MPH

The FDA accepted fenebrutinib's new drug application under priority review for relapsing and primary progressive MS, based on 3 phase 3 trials comparing the BTK inhibitor with teriflunomide and ocrelizumab.

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.

PETUNIA Study to Track Nipocalimab Use in Pregnancy Marco Meglio

PETUNIA, a new global pharmacovigilance study, will track pregnancy, maternal, and infant outcomes among patients with gMG exposed to nipocalimab.

Vanda to Pursue sNDA for Tasimelteon in Delayed Sleep-Wake Phase Disorder After Positive Phase 3 Readout Isabella Ciccone, MPH

In a pivotal phase 3 study, once-daily tasimelteon 20 mg shifted sleep onset 37.1 minutes earlier than placebo in adults with delayed sleep-wake phase disorder.

Single Infusion of Rese-Cel Yields Meaningful Responses Off Immunomodulators in Phase 1/2 RESET-MG Trial Isabella Ciccone, MPH

Findings from the phase 1/2 RESET-MG trial, presented at AANEM 2026, showed that 10 of 13 patients with refractory generalized myasthenia gravis had clinically meaningful MG-ADL improvements after a single infusion of rese-cel.

Todays Updates

IZAR-1: Sonelokimab Meets Phase 3 End Points for PsA

IZAR-1: Sonelokimab Meets Phase 3 End Points for PsA

What 2 Seasons of Arexvy Data Reveal About RSV-Related Risk Reduction Ann Falsey, MD

Two-season real-world data show Arexvy reduces RSV-related MACE and COPD and asthma exacerbations in adults 60 and older, Ann Falsey, MD, says.

Dupilumab Outperforms Omalizumab for Asthma Remission in Phase 4 EVEREST Guy Brusselle, MD, PhD

Guy Brusselle, MD, PhD, says that 64% of dupilumab-treated patients met composite asthma clinical remission criteria at week 24 versus 40.5% with omalizumab.

What The FDA Psychedelic Hearing Means for Psychiatrists

What The FDA Psychedelic Hearing Means for Psychiatrists

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.

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 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.

Todays Updates

IZAR-1: Sonelokimab Meets Phase 3 End Points for PsA

IZAR-1: Sonelokimab Meets Phase 3 End Points for PsA

What 2 Seasons of Arexvy Data Reveal About RSV-Related Risk Reduction Ann Falsey, MD

Two-season real-world data show Arexvy reduces RSV-related MACE and COPD and asthma exacerbations in adults 60 and older, Ann Falsey, MD, says.

Dupilumab Outperforms Omalizumab for Asthma Remission in Phase 4 EVEREST Guy Brusselle, MD, PhD

Guy Brusselle, MD, PhD, says that 64% of dupilumab-treated patients met composite asthma clinical remission criteria at week 24 versus 40.5% with omalizumab.

What The FDA Psychedelic Hearing Means for Psychiatrists

What The FDA Psychedelic Hearing Means for Psychiatrists

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.

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 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.

Latest News

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

The FDA accepted fenebrutinib's new drug application under priority review for relapsing and primary progressive MS, based on 3 phase 3 trials comparing the BTK inhibitor with teriflunomide and ocrelizumab.

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

PETUNIA, a new global pharmacovigilance study, will track pregnancy, maternal, and infant outcomes among patients with gMG exposed to nipocalimab.

In a pivotal phase 3 study, once-daily tasimelteon 20 mg shifted sleep onset 37.1 minutes earlier than placebo in adults with delayed sleep-wake phase disorder.

Findings from the phase 1/2 RESET-MG trial, presented at AANEM 2026, showed that 10 of 13 patients with refractory generalized myasthenia gravis had clinically meaningful MG-ADL improvements after a single infusion of rese-cel.

News All Time

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

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.

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.

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.

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.

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.

CAR-T Agent KYV-101 Show Durable Remission of Generalized Myasthenia Gravis in 1-Year Data Marco Meglio

One-year KYSA-6 data showed KYV-101, a CD19 CAR T-cell therapy, produced durable, drug-free responses in generalized myasthenia gravis.

NeurologyLive® Friday 5 — September 25, 2026 Isabella Ciccone, MPH

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 25, 2026.

Cognitive Dysfunction in MS: Rethinking Assessment and Management Lindsay Ross, MD, Rachel Galioto, PhD

MS Compass: Guiding Clinical Care, a podcast hosted by neuroimmunologist Lindsay Ross, MD, brings you an exclusive interview with Rachel Galioto, PhD. [LISTEN TIME: 33 minutes]

Navigating a Career in Neurology Through Mentorship, Exploration, and Medical Education Louie Pasculli, Blake Buletko, MD, Galina Gheihman, MD

A. Blake Buletko, MD, and Galina Gheihman, MD, discuss their career paths in neurology, the role of mentorship and resilience, and strategies for navigating professional growth as early-career clinician-educators.

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