Neuromuscular

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.

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.

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.

Neurology News Network. for the week ending October 26, 2024. [WATCH TIME: 4 minutes]

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

The director of the myasthenia gravis clinic at Yale University provided additional insight on the MINT study of inebilizumab in myasthenia gravis, some of the subanalyses within, and next plans in the drug’s development. [WATCH TIME: 5 minutes]

The chief medical officer at Edgewise Therapeutics gave an overview of the mechanism of EDG-5506, an agent in development for Becker muscular dystrophy, and its early promising clinical results to date. [WATCH TIME: 6 minutes]

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.

ArthemiR, a placebo-controlled study, primarily evaluates the safety of ATX-01 in myotonic dystrophy type 1, with other assessments that include target engagement and measures associated with muscle function.

The senior scientist at Sunnybrook Research Institute in Toronto, Ontario, provided clinical insight on his lecture given at AANEM 2024, focusing on the challenges and opportunities of teaching the next generation of practitioners.

With the positive recommendation, it marks a key step toward eplontersen’s availability in Europe as a treatment for ATTR polyneuropathy.

Constantine Farmakidis, MD, an associate professor of neurology at the University of Kansas Medical Center, provided clinical commentary on a subanalysis of a phase 3 study assessing nipocalimab, an investigational agent, in generalized myasthenia gravis.

Study investigator Henry Kaminski, MD, provided clinical insight on a unique trial assessing the efficacy and safety of oral cladribine tablets, an FDA-approved medication for multiple sclerosis, in patients with myasthenia gravis.

The data shows that changes in neuropathy impairment scores using nerve conduction studies may forecast which patients will respond better to treatment.

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

A recent analysis of the phase 3 ADHERE trial demonstrated the clinical benefit of subcutaneous efgartigimod PH20 in patients with chronic inflammatory demyelinating polyneuropathy.

Tuan Vu, MD, a professor of neurology at the University of South Florida, provided brief commentary on topline data of the phase 3 VIVACITY-MG3 study presented at AANEM 2024.

The phase 3 study is expected to include 110 patients with seronegative myasthenia gravis who will be randomly assigned to IV efgartigimod or placebo for a 5-week follow-up, followed by an open-label extension.

Neurology News Network. for the week ending October 19, 2024. [WATCH TIME: 4 minutes]

A new analysis revealed zilucoplan’s long-term efficacy and safety in patients with generalized myasthenia gravis, with data extending to 120 weeks of treatment.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 18, 2024.