Sponsored by Novartis, the RESTORE registry collects data from patients with spinal muscular atrophy (SMA), in particular on one of three treatments (Zolgensma, Spinraza and Evrydsi). Data from 168 patients treated with Zolgensma from this registry were analyzed:
- patients were aged between 0 and 6 months at diagnosis, and treated between one and 10 months of age,
- 47.6% carried two copies of the SMN2 gene and 41.7% three copies,
- motor performance was maintained or improved during 14 months of follow-up,
- 58.3% were identified by neonatal screening and had a better CHOP INTEND score than those diagnosed at a symptomatic stage,
- side effects were the same as those usually observed with Zolgensma.
