Cost-effectiveness of treatments for presymptomatic newborn patients with spinal muscular atrophy and two or three copies of the survival motor neuron 2 gene in Italy
Eur J Health Econ. 2025 Jun 26. doi: 10.1007/s10198-025-01806-2. Online ahead of print. ABSTRACT OBJECTIVE: We assessed the cost effectiveness of onasemnogene abeparvovec (OA) for …