Neuromuscular Trials

Is PrimeC (celecoxib + ciprofloxacin), a fixed-dose combination targeting neuroinflammation, iron homeostasis, and microRNA dysregulation, safe and well tolerated in people with ALS, and does it show signals of clinical and biomarker efficacy?

How accurate is muscle ultrasonography at detecting fasciculations for diagnosing amyotrophic lateral sclerosis?

Can an adaptive platform trial design provide operational advantages for testing multiple ALS therapeutics concurrently?

Can a single infusion of BCMA-targeted CAR-T cells produce durable improvement in generalized myasthenia gravis?

Is vamorolone safe and well tolerated in older (7- to <18-year-old) boys with DMD, including those switching from classic corticosteroids, and how does it affect growth, bone biomarkers, and adrenal function?

Does a single IV dose of delandistrogene moxeparvovec slow disease progression over 2 years in ambulatory boys with DMD?

Does givinostat slow disease progression in ambulant boys with DMD on corticosteroids?

In patients with spinal muscular atrophy, does high-dose nusinersen (50-mg loading, 28-mg maintenance) improve motor outcomes more rapidly than standard-dose nusinersen or sham control?

Does adding amifampridine modified release to pyridostigmine improve symptoms in patients with AChR-positive myasthenia gravis whose symptoms are insufficiently controlled on pyridostigmine alone?

In generalized AChR antibody-positive myasthenia gravis, how do complement inhibitors and FcRn blockers compare in efficacy and safety?

Is satralizumab, an IL-6 receptor inhibitor, safe and effective for treating seropositive generalised myasthenia gravis compared to placebo?

Can complement C5-targeted therapies — cemdisiran siRNA, pozelimab, or their combination — improve outcomes in generalised myasthenia gravis compared with placebo?

Does inebilizumab, a CD19+ B-cell–depleting monoclonal antibody, improve symptoms and function in patients with autoimmune generalized myasthenia gravis who are positive for anti–AChR or anti–MuSK antibodies compared with placebo?