A registry-based cohort study of Spinal Muscular Atrophy (SMA) disease to describe the natural history of SMA, the evolution of SMA care management and disease progression considering new disease modifying therapies (DMTs).

First published 27/01/2023

Last updated 04/05/2024

EU PAS number:

EUPAS50476

Study

Ongoing

Documents

Study protocol

Initial protocol

Protocol_EMA_SMA Version 1.3 Signed March 3 2023 (1)

English (1.51 MB - PDF)View document

Study results

Study report

SIGNED-SMA EMA - Report - FINAL- April 5 2024 + Appendix D-E-F.docx_.pdf

English (9.26 MB - PDF)View document

Other information

Study, other information

Objective 0 (Preliminary) Results

English (589.55 KB - PDF)View document

Objective 1 (Natural History) Results

English (1.07 MB - PDF)View document

Objective 2 (Healthcare) Results

English (224.62 KB - PDF)View document

Supplementary Results Objective 1 (Natural History)

English (8.15 MB - PDF)View document

Supplementary Results Objective 2 (Healthcare)

English (508.97 KB - PDF)View document

ENCePP Seal

ENCePP Seal documents

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