Figure 1.

Treatment and assessment schedule. We began treatment shortly after a preparational day hospital evaluation. Within the first 2 months of treatment, four administrations of Nusinersen take place within the ‘loading phase’. The treatment has to be repeated every 4 months thereafter. Intrathecal injections of Nusinersen were conducted by neurologists and neuroradiologists, the assessments involved neurologists, physical therapists, the pulmonary unit, and the laboratory for blood and urine workup.