Neuromuscular Trials

Vemircopan, an oral factor D inhibitor, did NOT improve MG-ADL or other efficacy endpoints versus placebo in AChR-Ab+ generalized MG, and the phase 2 trial was terminated early for lack of efficacy de...

In 13 RCTs (834 patients) with DPN, vitamin B improved MNSI clinical scores and sural sensory nerve conduction, but did not consistently reduce generic pain or motor nerve parameters; substantial hete...

Pregabalin monotherapy is non-inferior to pregabalin + ALA combination for pain relief in painful DPN over 12 weeks, with comparable safety; combination therapy may offer added benefit only in patient...

FcRn inhibitors, C5 complement inhibitors, and CD19+ B-cell depletion therapy show comparable efficacy on QMG and MG-ADL reduction in gMG (differences between classes below minimum clinically importan...

Pooled 1-year data from 4 studies (302 boys) show that delandistrogene moxeparvovec produces small but statistically significant improvements in NSAA, time to rise, and dystrophin expression versus st...

In 20 corticosteroid-naive boys aged 2-<4 years with DMD, 12 weeks of vamorolone (2 or 6 mg/kg/d) was well tolerated with no serious adverse events and stable growth, dose-dependent adrenal suppressio...

Over a median 4.9-year follow-up, earlier tofersen was associated with numerically less decline in ALSFRS-R, SVC, HHD strength, and quality-of-life measures and substantial sustained reductions in pla...

Do repeated 30-minute high-concentration capsaicin (8%) topical system treatments improve peripheral sensory function in patients with painful diabetic peripheral neuropathy with baseline sensory deficits?

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?

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?

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?

In generalized AChR antibody-positive myasthenia gravis, how do complement inhibitors and FcRn blockers compare with placebo/standard care in efficacy and safety, and do they differ in durability?

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?

Does Descartes-08, an autologous BCMA-directed mRNA CAR T cell therapy administered as six once-weekly outpatient infusions without preconditioning chemotherapy, improve clinical outcomes compared to placebo in patients with generalized myasthenia gravis?

Can six once-weekly outpatient infusions of BCMA-directed mRNA CAR-T therapy, without preconditioning chemotherapy, produce durable improvement in generalized myasthenia gravis?

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

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?