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Olipudase Alfa: First Approval
Drugs, 2022 Olipudase alfa (XENPOZYME®) is a recombinant human acid sphingomyelinase that has been developed by Sanofi, for the treatment of acid sphingomyelinase deficiency (ASMD). Olipudase alfa catalyses the hydrolysis of sphingomyelin accumulated in hepatocytes and in mononuclear-macrophage cells, such as the lungs, liver, spleen, kidneys and bone marrow ...openaire +4 more sourcesTwo-year results of the ASCEND trial of olipudase alfa adults with chronic acid sphingomyelinase deficiency show parallel improvements in former placebo patients and further improvement in continuing olipudase alfa patients
Molecular Genetics and Metabolism, 2022 Melissa P. Wasserstein, Antonio Barbato, Renata C. Gallagher, Roberto Giugliani, Norberto B. Guelbert, Julia B. Hennermann, Carla Hollak, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth L. Thurberg, Abhimanyu Yarramaneni, Andreea M. Rawlings, Yong Kim, Monica Kumar +20 moreopenaire +1 more sourceReversal of interstitial lung disease after olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency
Rare ILD/DPLD, 2023 Bonella, Francesco, Wasserstein, Melissa, Giugliani, Roberto, Lachmann, Robin, Noth, Imre, Scarpa, Maurizio, Wuyts, Wim A., Aguiar, Mario, Armstrong, Nicole M., Jessel, Andreas, Kumar, Monica, Waters, Sara, Berger, Kenneth I. +12 moreopenaire +1 more source
