Track 06 of 9
Neuromuscular Disease
ALS, SMA gene therapy and neuropathies.
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.
Focus areas
- SMA: nusinersen, onasemnogene abeparvovec, risdiplam comparative outcomes
- Duchenne: Elevidys gene therapy, exon-skipping ASOs, givinostat
- ALS: tofersen for SOD1, AMX0035 PHOENIX, C9orf72 and FUS ASOs
- Neurofilament light as a trial endpoint and accelerated-approval pathway
- Myasthenia gravis: FcRn antagonists (efgartigimod, rozanolixizumab) and C5 inhibitors
- CIDP and autoimmune neuropathies: nodal/paranodal antibodies
- Charcot-Marie-Tooth and inherited neuropathy gene-therapy pipeline