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Clinical Trials in Rare Disease: Challenges and Opportunities
The neuronal ceroid lipofuscinoses constitute one of many groups of rare childhood diseases for which disease-modifying treatments are non-existent. Disease-specific barriers to therapeutic success include incomplete understanding of disease pathophysiology and limitations of treatments that cannot...
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| 主要な著者: | , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2013
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3964003/ https://ncbi.nlm.nih.gov/pubmed/24014509 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0883073813495959 |
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