Real-world data collection in Niemann-Pick disease type C: Data from expanded access program with arimoclomol
| Autor: | Berry-Kravis, Elizabeth, Gallo, Daniel, Pagano, Kristin, Donnelly, Conan B. |
|---|---|
| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
| Externí odkaz: |
|
Full Text from ScienceDirect