Sometimes they come back: New and old spinal muscular atrophy adults in the era of nusinersen.

V A SansoneG CorattiM C PeraM PaneS MessinaF SalminE AlbamonteR De SanctisM SframeliV Di BellaS MorandoA d'AmicoA L FrongiaL AntonaciAlice PirolaM PedemonteEnrico Silvio BertiniC BrunoEugenio Mercurinull null

Published in: European journal of neurology (2020)

Our results showed differences between the two subgroups, even if less marked in the type III patients. In the type II patients, there was a higher proportion of newcomers who were in the severe end of the spectrum. Of the newcomers, only approximately a third initiated treatment, as opposed to the 51% in the regularly followed patients. The identification of patients who were not part of the registries will help to redefine the overall prevalence of SMA and the occurrence of different phenotypes.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
prognostic factors
peritoneal dialysis
risk assessment
type iii
early onset
patient reported outcomes
patient reported
combination therapy