Neuromuscular

NGN-401, an AAV9 gene therapy in development for Rett syndrome, was considered well-tolerated, with treated patients showing consistent gains in skill and developmental milestones.

The senior scientist at Sunnybrook Research Institute in Toronto, Ontario, provided clinical insight on the advantages of a flipped classroom, and why neurology educators should consider this approach. [WATCH TIME: 2 minutes]

Italo Linfante, MD, director of interventional neuroradiology at Baptist Health Miami Neuroscience Institute, discussed the importance of brain aneurysms in the context of neurology.

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

Judith Thompson, PharmD, MPH, CPHQ, rare disease population health strategy lead at UCB, discussed the association between social determinants of health and delayed diagnosis/misdiagnosis in myasthenia gravis.

Catch up on any of the neurology news headlines you may have missed over the course of October 2024, compiled all into one place by the NeurologyLive® team.

The double-blind, placebo-controlled trial will include 200 patients with Angelman syndrome who will be followed over a 12-month period, with an additional open-label extension after.

Neurology News Network. for the week ending November 9, 2024. [WATCH TIME: 4 minutes]

Despite not meeting its primary end point, treatment with GV1001 showed more promising results in patients with progressive supranuclear palsy-Richardson’s syndrome over a 24-week treatment period.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 8, 2024.

In previously reported data, AOC 1020 demonstrated a consistent reduction in double homeobox 4 regulated genes among patients with facioscapulohumeral muscular dystrophy at 4 months.

Diego Torres-Russotto, MD, chair of neurology at Baptist Health Miami Neuroscience Institute, discussed the Miami Neuroscience Symposium’s focus on addressing the community’s neurological needs, balancing content delivery and fostering multidisciplinary collaboration.

Michael McDermott, MD, chief medical executive at Baptist Health Miami Neuroscience Institute, gave greater insights on the institute’s annual symposium, which offers attendees updates in cutting-edge imaging, surgical techniques, and neurocognitive research.

Michael McDermott, MD, chief medical executive at Baptist Health Miami Neuroscience Institute, gave greater insights on the institute’s annual symposium, which offers attendees updates in cutting-edge imaging, surgical techniques, and neurocognitive research.

The director of the myasthenia gravis clinic at Yale University discussed the therapeutic potential of inebilizumab, an FDA-approved treatment for NMOSD, in myasthenia gravis, based on data from the phase 3 MINT study.

The NDA includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.

The director of the Muscular Dystrophy Clinic at UCSF Benioff Children’s Hospital provided insight on promising data from the phase 2/3 VIBRANCE-MG study assessing investigational nipocalimab in adolesents with myasthenia gravis. [WATCH TIME: 3 minutes]

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

Amy Tsou, MD, MSc, program director at the National Institutes of Neurological Disorders and Stroke, discussed the increasing cost of prescription drugs in the US and its impact on healthcare providers, including those who treat patients with neurologic conditions.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 1, 2024.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Lawrence Robinson, MD. [LISTEN TIME: 15 minutes]

The rare disease population health strategy lead at UCB provided clinical insight on some of the unique challenges of diagnosing myasthenia gravis, and some of the early signs clinicians and non-specialists should look out for. [WATCH TIME: 3 minutes]

Melissa Spencer, PhD, gave commentary on her presentation from AANEM 2024, highlighting the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies.

Prior to the 2025 MDA Conference, the vice president of Public Policy & Advocacy at the Muscular Dystrophy Association talked about the increasing interest in gene therapy in the field of neuromuscular diseases.

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