Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
| Title: | Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy |
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| Authors: | Jones, Simon A., McGovern, Margaret, Lidove, Olivier, Giugliani, Roberto, Mistry, Pramod K., Dionisi-Vici, Carlo, Munoz-Rojas, Maria-Veronica, Nalysnyk, Lubomyra, Schecter, Alison D., Wasserstein, Melissa |
| Source: | In Molecular Genetics and Metabolism September-October 2020 131(1-2):116-123 |
| Database: | ScienceDirect |
| ISSN: | 10967192 |
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| DOI: | 10.1016/j.ymgme.2020.06.008 |
| Published in: | Molecular Genetics and Metabolism |
| Language: | English |