Neuromuscular

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.

The assistant professor of neurology at Wake Forest School of Medicine provided clinical insight on MG0020, a phase 3 trial assessing a self-administration of rozanolixizumab through a syringe driver. [WATCH TIME: 3 minu]

Despite balanced baseline characteristics, patients ranged from low to high number of rozanolixizumab treatment cycles, suggesting the need for a personalized approach.

Findings showed that both fixed cycles and every-other-week dosing regimens of efgartigimod were well tolerated and effective in improving clinical outcomes in patients with generalized myasthenia gravis.

The PREVAIL trial, presented at the 2024 AANEM meeting, is investigating gefurulimab, a bispecific nanoantibody designed to inhibit complement activation, as a potential treatment for patients with generalized myasthenia gravis.

The neuromuscular specialist at the University of Tennessee Medical Center provided clinical insight on a retrospective analysis studying the long-term corticosteroid patterns of approved treatments for generalized myasthenia gravis. [WATCH TIME: 5 minutes]

Rozanolixizumab demonstrated efficacy in patients with generalized myasthenia gravis aged at least 65 years, despite a higher incidence of comorbidities observed in this population.

The Meta A. Neumann Professor of Neurology at George Washington University provided an overview of a new phase 3 study testing the efficacy and safety of cladribine versus placebo in generalized myasthenia gravis. [WATCH TIME: 3 minutes]

Pozelimab and cemdisiran work together to block the complement pathway, which plays a key role in gMG, using two different approaches—one suppresses liver production of a key protein, while the other targets it directly with antibodies.

Zilucoplan, approved for generalized myasthenia gravis in 2023, showed consistent complement inhibition, even with additional treatments like IVIg or plasma exchange.

The director of the myasthenia gravis clinic at Yale University provided clinical insight on promising phase 3 data assessing inebilizumab, an FDA-approved therapy for NMOSD, in patients with myasthenia gravis. [WATCH TIME: 4 minutes]

A new analysis of the phase 3 PROPEL study showed that switching to cipaglucosidase alfa/miglustat therapy enhanced outcomes among patients with late-onset Pompe disease.