News

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

Despite facing mental health concerns with anger, aggression, or irritability, among others, slightly less than one-fourth of patients with DMD utilized psychosocial services such as counseling or therapy.

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 on the MDA Clinical & Scientific conference.

The phase 2 CANYON trial findings presented at the 2025 MDA conference highlight sevasemten’s potential in reducing muscle injury biomarkers in Becker muscular dystrophy.

Neurology News Network. for the week ending March 22, 2025. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 21, 2025.

A duo of experts from Weill Cornell Medicine talked about the advancements made in diffuse midline gliomas research and clinical trials that may offer new hope for improving its historically poor prognosis.

The professor of pediatrics and division chief of Medical Genetics at Duke University gave clinical insight on a presentation on the advancements and limitations of enzyme replacement therapy for Pompe Disease. [WATCH TIME: 3 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Robert Califf, MD. [LISTEN TIME: 9 minutes]

New biomarker data presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference of RGX-202 in the phase 1/2 trial highlight strong microdystrophin expression.

The medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed findings from phase 3 trials suggesting that baseline PRLs can predict disability accumulation and may serve as biomarkers for treatment response to tolebrutinib. [WATCH TIME: 6 minutes]

A new analysis reported that givinostat, an oral histone deacetylase inhibitor recently approved for DMD, slowed disease progression by approximately 2 years compared with standard care.

The professor of pediatrics and division chief of Medical Genetics at Duke University delved into the transformative impact of enzyme replacement therapy on Pompe disease, addressing its advancements, limitations, and promising innovations shaping its future. [WATCH TIME: 2 minutes]

Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, shared his powerful journey as a patient advocate and gene therapy pioneer, shedding light on the challenges and progress in LGMD.

The company recently announced positive topline results from its phase 3 study in patients with MG and initial data from its phase 2b study in patients with CIDP, both assessing batoclimab.

Two separate long-term extension studies met their pre-specified endpoints, showing highly significant evidence of sustained treatment benefits in slowing disease progression in pediatric and adult patients.

The design of a phase 2 trial to investigate the potential of satralizumab in boys with Duchenne muscular dystrophy was recently presented at the 2025 MDA Clinical & Scientific Conference.

The phase 3b STRENGTH study reported that a single dose of OAV101IT was well tolerated in treatment-experienced patients with spinal muscular atrophy, with motor function stabilizing over 52 weeks.

Real-world findings from the RESTORE registry presented at the 2025 MDA conference confirmed the established safety profile of onasemnogene abeparvovec over a 5-year period.

Patient advocate and MDA Ambassador Lily Sander shared inspiring perspective on Charcot-Marie-Tooth disease, advocacy efforts, and the importance of connection

Long-term follow-up data from an early-phase study of an AAV8-based gene therapy for DMD suggest the treatment was well-tolerated and provided sustained biochemical and functional benefits.

Alpha Cognition has officially launched its new treatment for mild to moderate Alzheimer disease, with a focus on expanding patient access in the long-term care market.

The chief scientific officer at Avidity Biosciences provided clinical perspective on the function and mechanism of del-zota, an investigational antisense treatment in development for DMD amenable to exon 44 skipping. [WATCH TIME: 3 minutes]

Findings from the phase 2 FIGHT DMD trial suggest an investigational oral thromboxane prostanoid receptor antagonist may preserve heart function in patients with DMD-associated cardiomyopathy.

This phase 4 study on omaveloxolone will assess long-term maternal and infant health, potentially informing future safety guidelines for patients with Friedreich ataxia.

The MyacarinG study post-hoc analysis demonstrated rozanolixizumab's potential to significantly reduce ocular symptoms in gMG, with consistent improvements across multiple scoring systems.

Three patients treated with DT-DEC01 showed improvements in various functional tests, including echocardiography, arm movements, grip strength, and spirometry after 12-24 months of treatment.

In the study, most participants are aged 18-60, with 53% female and 73.1% carrying the c.826C>A mutation, while 15 patients are aged 12-18.