Results 71 to 80 of about 285 (98)
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Olipudase Alfa: First Approval
Drugs, 2022Olipudase 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 ...
Susan Keam, Keam Susan J
exaly +3 more sources
First experience with olipudase alfa for ASMD type B in Mexico
Molecular Genetics and MetabolismLuz Maria Sanchez +1 more
exaly +2 more sources
Efficacy and safety of olipudase alfa in children: A real-life case report
Molecular Genetics and MetabolismFabiano De Oliveira Poswar +1 more
exaly +2 more sources
American Journal of Medical Genetics Part A
ABSTRACT Acid sphingomyelinase deficiency (ASMD), or Niemann–Pick disease types A, B, and A/B, is a rare lysosomal storage disorder caused by SMPD1 mutations. Clinical forms range from severe neurovisceral (type A) to chronic visceral (type B), mainly affecting the liver, spleen, and lungs.
Breno Bopp Antonello +6 more
openaire +2 more sources
ABSTRACT Acid sphingomyelinase deficiency (ASMD), or Niemann–Pick disease types A, B, and A/B, is a rare lysosomal storage disorder caused by SMPD1 mutations. Clinical forms range from severe neurovisceral (type A) to chronic visceral (type B), mainly affecting the liver, spleen, and lungs.
Breno Bopp Antonello +6 more
openaire +2 more sources