Article about Zolgensma® in Switzerland

Spinal muscular atrophy (SMA) is a severe neurodegenerative disease. For a few years now, SMA can be treated with genetic therapies. These new therapies include the gene therapy Zolgensma®. Zolgensma® has been approved in Switzerland for the treatment of children with SMA under the age of 2 since mid-2021. Even before approval, some patients could be treated with Zolgensma® gene therapy. We have now analyzed the treatment and follow-up data recorded in the registry for the first 9 children treated with Zolgensma® in Switzerland.

All children in this study showed motor development progress after treatment with Zolgensma®. This progress would not have been expected without the gene therapy. None of the children died during the observation period, which averaged just under 13 months. At the end of the observation period, some children required support with feeding and breathing in the form of additional tube feeding and/or nocturnal mask ventilation. In some children, temporary changes in laboratory values have been observed after gene therapy, for example a decrease in blood platelet counts or an increase in liver values. It is known that these laboratory changes can occur after gene therapy. No special therapeutic measures were necessary due to these side effects.

Our study shows that Zolgensma® is an effective treatment for SMA and has a particularly positive effect on the motor development of the treated infants and toddlers.

Link to the article: Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study