
Interim phase 1/2 data showed increased lean muscle volume and a favorable safety profile following a single dose of EPI-321 in the first three evaluable patients with facioscapulohumeral muscular dystrophy.

Louie Pasculli, Assistant Editor for NeurologyLive®, has been with the team since October 2025. Follow him on X @Louie_Pasculli or email him at [email protected]

Interim phase 1/2 data showed increased lean muscle volume and a favorable safety profile following a single dose of EPI-321 in the first three evaluable patients with facioscapulohumeral muscular dystrophy.

In the exploratory phase 2 RoAD study, PrimeC demonstrated biomarker changes consistent with target engagement across multiple neurodegenerative pathways, informing the design of future Alzheimer's disease trials.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is sex-based differences in neurology!

Movement disorder experts Jonathan Isaacson, MD, and Abhishek Lenka, MD, PhD, discuss early identification and accurate diagnosis of autonomic dysfunction in Parkinson disease, highlighting emerging biomarkers and monitoring tools that may enable more personalized care.

Following the FDA's expanded approval of efgartigimod, the neuromuscular physician at Erlanger Health Systems discusses the implications for seronegative disease, treatment sequencing, and the evolving definition of antibody-negative MG. [WATCH TIME: 5 minutes]

At ATMRD 2026, a neurologist at UCLA discussed the growing recognition of demoralization in Parkinson disease, highlighting how it differs from depression and apathy and outlining strategies to help patients maintain resilience, purpose, and quality of life. [Watch time: 5 minutes]

The 2026 Alzheimer disease drug development landscape highlights a growing shift toward diverse disease-modifying strategies, from amyloid and tau targeting to metabolic and neuroprotective approaches.

FDA’s clearance for PTD802, a first-in-class GPR17 antagonist designed to promote remyelination, enables initiation of a first-in-human phase 1 study in healthy volunteers and advances a novel regenerative approach for multiple sclerosis.

The associate professor of neurology and neuroscience at the Icahn School of Medicine at Mount Sinai discussed plasma p-tau217 levels association with regional hypometabolism, cortical atrophy, and cognitive impairment in early symptomatic Alzheimer disease.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is adaptive deep brain stimulation!

The phase 3 REVITALYZ study assessed whether once-nightly extended-release sodium oxybate can improve excessive daytime sleepiness and other core symptoms of idiopathic hypersomnia.

New preclinical findings suggest that SOM3355's combined VMAT1 inhibition, VMAT2 modulation, and β1-adrenergic antagonism may offer a differentiated approach to managing motor and neuropsychiatric symptoms in Huntington disease.

The FDA indicated that 3-year phase 1/2 data may support a BLA submission for AMT-130, an investigational gene therapy that previously demonstrated significant slowing of Huntington disease progression in treated patients.

AT ATMRD 2026, the CEO and founder of Yes, and... X discussed why social connection and support groups should be viewed as critical components of Parkinson disease care, alongside traditional medical management. [WATCH TIME: 3 minutes]

Long-term phase 3 extension data showed that pitolisant was generally well tolerated and associated with sustained improvements in excessive daytime sleepiness, sleep inertia, and functional outcomes in adults with idiopathic hypersomnia for more than 2 years.

The phase 3 EMNERGIZE study evaluates whether the complement C2 inhibitor empasiprubart can improve disability and functional outcomes in adults with chronic inflammatory demyelinating polyneuropathy.

A randomized, placebo-controlled study will assess whether weekly subcutaneous imeroprubart can reduce relapse risk in patients with chronic inflammatory demyelinating polyneuropathy despite standard-of-care treatment.

Neurologists Sarah Simmons, MD, PhD, and Fatma Inanici, MD, PhD, discuss how the combination of noninvasive cervical spinal cord stimulation with exercise therapy can improve upper extremity function in people with MS.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is ferroptosis!

The senior vice president for research at the Kessler Foundation discussed findings showing associations between reductions in neurofilament light chain and gains in cognitive processing speed, verbal memory, and visual memory in patients with early relapsing multiple sclerosis treated with ozanimod. [WATCH TIME: 3 minutes]

The professor of psychiatry and neurology reviewed approaches to treating chorea, cognitive impairment, apathy, irritability, and aggression in Huntington disease, while highlighting the promise of huntingtin-lowering therapies.

At ATMRD 2026, the director of the Parkinson's Disease and Movement Disorders Program at the Cleveland Clinic discussed the importance of movement phenomenology and accurate diagnosis in guiding treatment decisions for patients with hyperkinetic disorders. [WATCH TIME: 7 minutes]

The professor at MedStar Georgetown University Hospital spoke about a live session that featured patient evaluations and injection demonstrations designed to help clinicians refine treatment strategies for dystonia, spasticity, sialorrhea, and other neurologic conditions. [WATCH TIME: 2 minutes]

At ATMRD 2026, the director of Movement Disorders at Banner Sun Health Research Institute discussed emerging biomarker and imaging technologies, the importance of clinical diagnostic expertise, and the evolving treatment landscape for atypical parkinsonian disorders and adult-onset ataxias.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is Brain insulin resistance!

At CMSC 2026, the neuroimmunologist and director at OhioHealth MS Center, discussed results from the phase 3 FENhance 1 and 2 studies, highlighting significant reductions in relapse rates and robust MRI outcomes. [WATCH TIME: 3 minutes]

As the MS community reflects on World MS Day, Alla Wilson, a clinical ambulatory pharmacist at Shields Health Solutions, discusses how integrated specialty pharmacy models can improve treatment access, adherence, and patient-reported outcomes for multiple sclerosis.

12-montn interim findings from an ongoing phase 1/2 study showed up to 69% reductions in mutant huntingtin protein and favorable cUHDRS trends with the investigational oral RNA-splicing modifier SKY-0515.

A clinical research coordinator at SunnyBrook Health Sciences Centre, explained key factors of the computerized cognitive training program that contributed to cognitive improvement. [WATCH TIME: 3 minutes]

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is mmoldering inflammation in MS!