The laboratory (CORTILAB) directed by Prof. Stefania Corti is one of the research facilities of the Dino Ferrari Center and the University of Milan (UNIMI) and is based in the Sforza and Invernizzi buildings at the Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan
Onasemnogene abeparvovec in spinal muscular atrophy
Predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies.
Our paper describes OA 12-month follow-up showing efficacy across various age and weight groups not targeted by clinical trials. The study identifies prognostic factors for safety and efficacy in treatment selection.