2026 marks a remarkable decade since the regulatory approval of the very first disease-modifying therapy (nusinersin) for spinal muscular atrophy (SMA) in 2016. In this episode with Josefine Gibson, Professor Michelle Farrar and Dr Sharika Raga from Sydney Children's Hospital share their perspectives on how diagnostic and therapeutic advances for SMA have transformed the lives of children and families affected by SMA. We discuss the new long-term efficacy and safety results from the 3-year extension of the international FIREFISH trial of risdiplam for symptomatic infants with type 1 SMA, and we explore what children in the real world who are in treatment for SMA are revealing about adaptive functioning in their daily lives. Coupled with newborn screening, disease modifying therapies are yielding significantly better longer-term outcomes for children affected by SMA, so we consider their changing and individualised health-care needs and top priorities for clinical research in the next decade.

Click here to read the full articles:

https://www.thelancet.com/journals/lanchi/article/PIIS2352-4642(26)00127-6/fulltext

https://www.thelancet.com/journals/lanchi/article/PIIS2352-4642(26)00126-4/fulltext

https://www.thelancet.com/journals/lanwpc/article/PIIS2666-6065(26)00070-2/fulltext