Neuromuscular Disease

Neuromuscular medicine has been reshaped by genetically targeted therapies: nusinersen, onasemnogene abeparvovec and risdiplam in SMA, exon-skipping ASOs and Elevidys gene therapy in Duchenne, and tofersen for SOD1-ALS following the VALOR trial and accelerated approval. GCNN 2027 will assess the mixed signal for AMX0035 after PHOENIX, the post-marketing experience with tofersen and neurofilament-driven trial design, and the next wave of ALS programs targeting C9orf72, FUS, and ATXN2. The track also covers CIDP (efgartigimod, rozanolixizumab), myasthenia gravis FcRn and complement inhibitors, and CMT gene-therapy programs.