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RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design

Published 17 November 2021

This registry will assess outcomes in patients with genetically confirmed SMA and provide information on the effectiveness and long-term safety of approved and emerging treatments.

Read the paper at https://content.iospress.com/articles/journal-of-neuromuscular-diseases…

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17 Nov 2021