Spinraza® (nusinersen) is a drug for the treatment of children and adults with spinal muscular atrophy (SMA). In recent years, clinical studies have shown that Spinraza® can improve muscle strength and motor function. With our study we aimed to find out whether Spinraza® also works when it is used in older, more severely or mildly affected people than was investigated in the clinical trials.
In Switzerland, about 60 people are currently being treated with Spinraza®. We investigated whether Spinraza® affects the motor skills of people with SMA. In our study, Spinraza® worked mainly in children, but also in many adolescents and adults. Very few people had side effects due to the treatment, and in most cases these were mild. Spinraza® is therefore an effective treatment not only in clinical trials, but also when used in a broad patient population.