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RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design.
Finkel, Richard S.; Day, John W.; De Vivo, Darryl C. et al.
2020 • In Journal of Neuromuscular Diseases, 7 (2), p. 145-152

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Restore_A prospective multinational registry of patients with genetically confirmed spinal muscular atrophy_Rationale and study design.pdf

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