Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials

This study retrospectively merged databases from different large multicentric networks in the United States and Europe in order to establish (i) 12-month HFMSE changes in a larger cohort than those previously reported; (ii) possible differences of progression between ambulant and non-ambulant patients; (iii) the possible effect of different variables, such as gender, baseline values or age on the progression of the disorder; and (iv) possible outliers and the reasons for performing differently. The results suggest that there are different profiles of progression in ambulant and non-ambulant patients, and that age may play an important role in the progression of non-ambulant patients.

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Mercuri E, Finkel R, Montes J, et al. Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials. Neuromuscul Disord. 2015 Dec 3. pii: S0960-8966(15)30056-0.