Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
Simon A. Jones, Margaret McGovern, Olivier Lidove, Roberto Giugliani, Pramod K. Mistry, Carlo Dionisi-Vici, Maria Veronica Munoz-Rojas, Lubomyra Nalysnyk, Alison D. Schecter, Melissa Wasserstein
Research output: Contribution to journal › Review article › peer-review
25Scopus
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