An international consortium of clinicians with expertise in SMA reports new natural history data in this disease:
- the study population consisted of 226 patients with SMAII and 162 with type III, from five countries (Belgium, Italy, Spain, the United States and the United Kingdom),
- change over time in the Hammersmith Modified Functional Scale (HFMSE) was used as the primary outcome measure, with the study taking place over four years,
- patients with type II SMA lost an average of 2.20 points on the HFMSE over the period, compared with 2.75 points for patients with type III SMA,
- the authors were able to identify age subgroups in which these variations were more relevant.
These natural history data will be useful for the design of future clinical trials in SMA.
