Spinal muscular atrophy (SMA) is the second most common neuromuscular disorder in children. There are 3 types (from I to III) depending on the age of onset of symptoms and the maximum functional abilities reached. Adult patients most often have SMA type II or III who have “aged” and much more rarely type I or IV. The recent arrival of innovative therapies based on knowledge of the SMN1 gene is changing the natural history of the disease provided they 
are administered at an early stage. However, the interest of nusinersen in particular still raises questions in adults, due to the lack, among other things, of a proper clinical trial, versus placebo, in this patient population.
Three articles recently published in March and April 2020, including an editorial, provide some answers. The national network of German myologists involved in SMA was able to demonstrate effectiveness by retrospectively analyzing data from 139 adult patients treated with nusinersen (aged 16 to 65). With a follow-up duration ranging from 6 to 14 months, this resulted in an improvement in the scores on the modified Hammersmith scale. Another German study, with a smaller cohort (7 patients) and a shorter follow-up (10 months) does not allow us to conclude in terms of effectiveness but emphasizes the feasibility and the good tolerance of the treatment.
