| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 8343054 | Molecular Genetics and Metabolism | 2018 | 7 Pages |
Abstract
The Blind Start study and MDRI design improve statistical power that enhances detection of a positive treatment effect in this rare heterogeneous disease and could be utilized for other ultra-rare diseases.
Keywords
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Authors
Paul Harmatz, Chester B. Whitley, Raymond Y. Wang, Mislen Bauer, Wenjie Song, Christine Haller, Emil Kakkis,