Study design challenges and strategies in clinical trials for rare diseases: Lessons learned from pantothenate kinase-associated neurodegeneration
Substantial challenges in study design and methodology exist during clinical trial development to examine treatment response in patients with a rare disease, especially those with predominant central nervous system involvement and heterogeneity in clinical manifestations and natural history. Here we...
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| Principais autores: | , , , , , , , |
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| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
Frontiers Media S.A.
2023-03-01
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| 叢編: | Frontiers in Neurology |
| 主題: | |
| 在線閱讀: | https://www.frontiersin.org/articles/10.3389/fneur.2023.1098454/full |
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