ALS

ALS treatment has been transformed by ASO and gene therapies, marking the first wave of disease-modifying interventions. The session covers tofersen (Qalsody) for SOD1 ALS with the VALOR open-label extension data, ulefnersen antisense ASO, C9orf72-targeted programmes (BIIB078 lessons, newer-generation candidates), the AMX0035 (Relyvrio) withdrawal after PHOENIX trial failure and lessons for accelerated approval, palliative ALS care principles, and the ALS Functional Rating Scale revised (ALSFRS-R) outcomes. Discussion addresses fasudil for sporadic ALS, IPS-cell-based therapy approaches, ALS in pediatric and young-adult populations, post-discharge home ventilation, multidisciplinary ALS clinics, and the role of NfL as a treatment-response biomarker.