Dutch and American resarchers have published an update to October 2018 of two Cochrane reviews concerning clinical trials of treatments able to stop or slow the progression of SMA type I, II or III. Previous editions (in 2009 and 2011) had not highlighted effective treatments in SMA; since then, nusinersen has been tested and has shown to be effective in SMA. However, gene therapy to replace the SMN1 gene (Zolgensma) has not been taken into account in this update.
Based on the critical analysis of 12 randomized controlled trials, 2 in SMA type I (nusinersen and riluzole) and 10 in SMA type II (nusinersen, olesoxime, creatine, phenylbutyrate, gabapentin, somatotropin, TRH (thyrotropin releasing hormon), hydroxyurea, valproic acid, association of valproic acid and L-carnitine),
The authors conclude that:
- nusinersen could extend overall survival or without ventilation and improve motor development in SMA type I;
- nusinersen improves motor function in SMA type II;
- none of the other treatments evaluated have been found to be effective in SMA type I, II or III.
