Neuromuscular

The SKYCLARYS PASS registry, an international observational cohort study, aims to include approximately 300 omaveloxolone-naïve patients participating in the Friedreich’s Ataxia Global Consortium's UNIFIED natural history study.

Investigators are set to test the effects of 300 mg/day fasudil after the therapy showed promising data on ALS-related biomarkers in 180 mg/day doses.

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

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 Friedreich ataxia.

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.

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.

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.

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.

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.

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

In a small sample population of adolescents with myasthenia gravis, nipocalimab met its primary end point, showing a significant reduction in total serum immunoglobuin over a 24-week period.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 25, 2024.

Fumihiko Urano, MD, PhD, principal investigator of the phase 2 HELIOS trial, discussed some of the notable findings from the study, as well as the potential of AMX0035 to treat patients with Wolfram syndrome.

The senior scientist at Sunnybrook Research Institute talked about both the opportunities and challenges with the shift of technological advancement in medical education, especially in fostering critical thinking and managing the vast influx of information. [WATCH TIME: 4 minutes]

Based on the positive data, the company plans to begin a phase 3 trial assessing FLT203 in patients with Gaucher disease type 1 in the second half of 2025.