Multivariate analysis of a longitudinal data mart for Spinal Muscular Atrophy. Retrospective study of patients affected by Spinal Muscular Atrophy based on Generator data clustering methods, to support clinical analysis of response to treatments

Target:

The primary objective of the study is to identify the influence of motor progression (e.g. orthopedic measures, functional scores), select biomarkers (e.g. laboratory data) and demographic information (sma type, age of onset, age at treatment etc.) on the disease progression; to analyze and classify patients’ groups accordingly, and to deliver other diagnostic and prognostic synthetic indicators. The results of this study will be the first-ever systematic approach to SMA, where predictive models for response to new treatments can be built and adopted in future studies and clinical practice.

This is a no-profit study co-sponsored by Roche Italia.