Treatment with Nusinersen – Challenges Regarding the Indication for Children with SMA Type 1

The natural history of patients with spinal muscular atrophy (SMA) has changed due to advances in standard care and development of targeted treatments. Nusinersen was the first drug approved for the treatment of all SMA patients. The transfer of clinical trial data into a real-life environment is c hallenging, especially regarding the advice of patients and families to what extent they can expect a benefit from the novel treatment. We report the...