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).

Study protocol

Initial protocol

Protocol_EMA_SMA Version 1.3 Signed March 3 2023 (1)

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Study results

Study report

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

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Other information

Study, other information

Objective 0 (Preliminary) Results

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Objective 1 (Natural History) Results

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Objective 2 (Healthcare) Results

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Supplementary Results Objective 1 (Natural History)

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Supplementary Results Objective 2 (Healthcare)

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