Neuromuscular Trials

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

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?

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

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

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

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

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

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?

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

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

BCMA-Targeted CAR-T Cell Therapy for Generalized Myasthenia Gravis

Does high-dose nusinersen provide greater motor improvement than standard-dose in infantile-onset SMA?

High-Dose Nusinersen for Spinal Muscular Atrophy

In non-ambulatory patients with type 2 or type 3 spinal muscular atrophy on background nusinersen or risdiplam, does add-on apitegromab (a selective myostatin inhibitor) improve motor function compared with placebo?

What is the safety, PK/PD profile, and preliminary efficacy of oral risdiplam in infants with SMA Type 1?

Does fordadistrogene movaparvovec, an rAAV9-based mini-dystrophin gene therapy, slow functional decline in ambulatory boys with Duchenne muscular dystrophy?

Is delpacibart etedesiran, an antibody-oligonucleotide conjugate targeting DMPK mRNA, safe and effective in reducing toxic DMPK RNA and improving clinical outcomes in adults with myotonic dystrophy type 1?

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 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?