Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
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, MelJournal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2020.06.008
Date:
June, 2020
File:
PDF, 712 KB
2020
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