RESTORE: a prospective multinational registry of patients with genetically confirmed spinal muscular atrophy - rationale and study design
<p><strong>Background:</strong> Dramatic improvements in spinal muscular atrophy (SMA) treatment have changed the prognosis for patients with this disease, leading to important new questions. Gathering representative, real-world data about the long-term efficacy and safety of emerg...
| 主要な著者: | , , , , , , , , , , , , , , , , |
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| フォーマット: | Journal article |
| 言語: | English |
| 出版事項: |
IOS Press
2020
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