Neuromuscular Trials

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

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 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?

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?

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

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?