The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
July 30th 2025
Capricor Therapeutics navigates FDA challenges for deramiocel, aiming to advance treatment for DMD-related cardiomyopathy with promising trial data.
Expert Insight on Pivotal Phase 2 Data of AMX0035 in Wolfram Syndrome
October 24th 2024Fumihiko 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.
Challenges and Opportunities of Adapting Medical Education in a Digital Age: Lawrence Robinson, MD
October 24th 2024The 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]
Patient Dosing Commenced for Phase 1/2 ArthemiR Trial of ATX-01 in Myotonic Dystrophy Type 1
October 23rd 2024ArthemiR, 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.
Understanding Nipocalimab’s Impact on Myasthenia Gravis Using MG-ADL
October 22nd 2024Constantine 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.
Details Behind the Phase 3 MyClad Study of Cladribine in Myasthenia Gravis
October 22nd 2024Study 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.
Phase 3 Trial to Assess Therapeutic Effect of IV Efgartigimod in Seronegative Myasthenia Gravis
October 19th 2024The 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.
Phase 3 PREVAIL Study to Test Bispecific Nanoantibody Gefurulimab in Generalized Myasthenia Gravis
October 17th 2024The 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.
Rozanolixizumab Efficacious in Older Patients With Generalized Myasthenia Gravis, Phase 3 Data Show
October 17th 2024Rozanolixizumab demonstrated efficacy in patients with generalized myasthenia gravis aged at least 65 years, despite a higher incidence of comorbidities observed in this population.
Phase 3 NIMBLE Trial to Test Effects of Pozelimab and Cemdisiran in Myasthenia Gravis
October 16th 2024Pozelimab 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.
Understanding the Therapeutic Potential of Inebilizumab in Myasthenia Gravis: Richard Nowak, MD, MS
October 16th 2024The 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]
Daily Oral Edaravone Fails to Display Superiority in Phase 3B Study MT-1186-A02 for ALS
October 15th 2024A new study presented at AANEM 2024 revealed that daily dosing of oral edaravone was well tolerated and did not reveal any new safety concerns in patients with amyotrophic lateral sclerosis.