To retrospectively investigate Safety and Efficacy of Nusinersen in a large cohort of adult Hungarian patients with Spinal Muscular Atrophy (SMA).

Abstract

Research for Safety and Efficacy for a new drug treatment (Nusinersen) for adult patients with SMA. six patients were participate, each one of them got five injections in total so far; in every meeting their muuscular status was evaluated through RULM tests and Hammersmith tests. Data were evaluated from the first injection and the fifth one. There is a significance difference on before and after results. Overall the treatment therapy is safe and positive for the patients.