The clinical spectrum of SMA-PME and in vitro normalization of its cellular ceramide profile

Michelle M. Lee, Graeme S.V. McDowell, Darryl C. De Vivo, Daniel Friedman, Samuel F. Berkovic, Maria Spanou, Argirios Dinopoulos, Katheryn Grand, Pedro A. Sanchez-Lara, Michelle Allen-Sharpley, Jodi Warman-Chardon, Alexander Solyom, Thierry Levade, Edward H. Schuchman, Steffany A.L. Bennett, David A. Dyment, Toni S. Pearson

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