Adults with chronic acid sphingomyelinase deficiency show significant visceral, pulmonary, and hematologic improvements after enzyme replacement therapy with olipudase-alfa: 1-year results of the ASCEND placebo-controlled trial

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Melissa Wasserstein, Laila Arash-Kaps, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto B. Guelbert, Carla Hollak, Takayuki Ikezoe, Robin Lachmann, Oliver Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Yixin Chen, Patricia A. Fraser, Beth L. Thurberg, Atef Zaher

Source: Molecular Genetics and Metabolism - February 2, 2021 Category: Genetics & Stem Cells Source Type: research