News

The FDA has recently granted approval to Soleno Therapeutics' DCCR tablets as the first treatment for hyperphagia in Prader-Willi syndrome.

WVE-N531, an investigational exon-skipping therapy, showed promising 48-week results in the FORWARD-53 study, marking the first-ever improvement in muscle health for patients with Duchenne muscular dystrophy, with plans for an NDA filing in 2026.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Stuart Isaacson, MD; and Rajesh Pahwa, MD. [LISTEN TIME: 17 minutes]

The HARBOR study is an ongoing phase 3 global study focused on assessing del‑desiran, formerly known as AOC 1001, which aims to treat the underlying cause of DM1.

The director of the pediatric epilepsy monitoring unit at the Medical University of South Carolina provided commentary on the significance of Purple Day as a means to raise awareness about epilepsy. [WATCH TIME: 3 minutes]

Emma Macdonald-Laurs, PhD, FRACP, MBChB, a neurologist at the Royal Children’s Hospital in Melbourne, Australia, provided commentary on the progress and challenges the epilepsy community faces amid Purple Day.

Luis Tornes, MD, a neurologist and director of Baptist Health’s Epilepsy Program, provided clinical insights on raising awareness for Purple Day, a global initiative dedicated for patients and families with epilepsy.

The senior vice president of research at The ALS Association discussed insights into neural regeneration therapies and their potential to restore function in neurodegenerative diseases.

The associate professor of pediatric neurology and genetics at the University of Alabama Birmingham provided clinical insights on the key signaling pathways currently being targeted in the treatment of muscular dystrophies. [WATCH TIME: 4 minutes]

Topline data from the REFOCUS-ALZ trial showed no significant cognitive or functional benefits with simufilam in patients with mild-to-moderate AD, leading the company to discontinue the program.

Neurizon Therapeutics announced updates to the HEALEY-ALS trial and promising NUZ-001 results, signaling a major step forward in ALS treatment research.

Tolebrutinib's potential approval as the first brain-penetrant BTK inhibitor for non-relapsing secondary progressive MS and to slow disability accumulation independent of relapse activity could represent a paradigm shift in treating disability driven by smoldering neuroinflammation.

The assistant professor in the department of neurology at Mount Sinai discussed distinguishing cognitive impairment in MS from AD emphasizing orientation as a key differentiator. [WATCH TIME: 5 minutes]

Pooled safety data of delandistrogene moxeparvovec, a gene transfer therapy for Duchenne muscular dystrophy, suggests a manageable tolerability profile up to 5 years of follow-up.

Findings from a phase 2 study of nomlabofusp, a novel investigational therapy for Friedreich ataxia, demonstrated an increase in tissue frataxin levels and metabolic improvements.

Despite advancements in NMOSD treatments, the founder and executive director of the Sumaira Foundation discussed how misdiagnosis, delayed diagnosis, and disparities in care remain significant challenges. [WATCH TIME: 4 minutes]

Results from a recent phase 3 study presented at the 2025 MDA Conference reported that oral edaravone remained well tolerated in patients with ALS over 96 weeks, with no new safety concerns.

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]

Marketed as Amvuttra, the Alnylam Pharmaceuticals RNA-interference treatment is now approved for an additional patient population of 150,000 in the US, and more than 300,000 worldwide.

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.