P.371Nusinersen treatment in spinal muscular atrophy: the experience of Bambino Ges ù Children's Hospital

In December 2016 and in June 2017, respectively, the FDA and EMA approved Nusinersen as the first treatment for SMA.Bambino Ges ù Hospital started to treat patients with the SMA type 1, from november 2016, as part of the expanded access programm (EAP), and from november 2017, after the commercial approval , the later onset forms. We recruited patients followed in the Neuromuscular Unit. We activated a multidisciplinary team composed of neurologists, pneumologists, anaesthesiologists, radiologists, physiotherapists and pharmacists, in order to prepare the drug, assess the motor functional abilities and monitor the effectiveness, assess the respiratory function and the risk of sedation and perform the injection in sever e scoliosis.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research