Patrícia Balážová

A multicenter, prospective – retrospective, epidemiological study in Czech and Slovak minors with spinal muscular atrophy treated with nusinersen

Abstract Background We used the Czech REaDY registry to collect and analyse data of paediatric patients with spinal muscular atrophy (SMA) who received nusinersen for five years. By sharing the results, we aim to increase healthcare professionals’ knowledge about this therapy. Re...