Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy

Publication date: Available online 24 June 2020Source: Molecular Genetics and MetabolismAuthor(s): 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
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research