Results 91 to 100 of about 166,938 (112)

Two-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 more
openaire   +1 more source

Olipudase alfa-Enzymersatztherapie bei ASMD

Gastro-News, 2021
openaire   +1 more source

EMA-Zulassung für Olipudase alfa

Gastro-News, 2022
openaire   +1 more source

Zulassung von Olipudase alfa als Enzymersatztherapie bei ASMD

Gastro-News, 2023
openaire   +1 more source

Reversal of Interstitial Lung Disease in Children and Adults With Chronic Acid Sphingomyelinase Deficiency Treated With Olipudase Alfa Enzyme Replacement Therapy

American Journal of Respiratory and Critical Care Medicine
Abstract Rationale: Adults and children with acid sphingomyelinase deficiency (ASMD), a rare autosomal recessive disease, have prominent pulmonary manifestations, hepatosplenomegaly, dyslipidemia, and growth deficits. Pathogenic variants in the SMPD1 gene result in deficient ASM activity, leading to progressive accumulation of ...
G. Raghu, M. Scarpa, R. Giugliani, R. Lachmann, M. Wasserstein, F. Bonella, I. Noth, W.A. Wuyts, N.M. Armstrong, M. Kumar, K.I. Berger   +10 more
openaire   +1 more source

Pediatric and adult acid sphingomyelinase deficiency (ASMD) patients exhibit mechanistic similarity in their disease and response to olipudase alfa

Molecular Genetics and Metabolism, 2023
Mengdi Tao, Randolph J. Leiser, Chanchala Kaddi, Bradley Niesner, Jing Li, Catherine Ortemann-Renon, Monica Kumar, Andreas Jessel, Susana Zaph   +8 more
openaire   +1 more source

Reversal 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 more
openaire   +1 more source

The impacts of olipudase alfa on adults with ASMD: The patient-reported experience

Molecular Genetics and Metabolism
Conan B. Donnelly, Solomon Mbua, Joslyn Crowe, Justin Hopkin   +3 more
openaire   +1 more source

First experience with olipudase alfa for ASMD type B in Mexico

Molecular Genetics and Metabolism
Luz Maria Sanchez, Magdalena Ceron Rodriguez   +1 more
openaire   +1 more source

One-year results of olipudase alfa enzyme replacement therapy in children with chronic visceral and neurovisceral acid sphingomyelinase deficiency

Molecular Genetics and Metabolism, 2021
George Diaz, Roberto Giugliani, Nathalie Guffon, Simon Jones, Eugen Mengel, Maurizio Scarpa, Isabela Batsu, Patricia Fraser, Jing Li, Qi Zhang, Catherine Ortemann-Renon   +10 more
openaire   +1 more source