Results 81 to 90 of about 285 (98)

Zulassung von Olipudase alfa als Enzymersatztherapie bei ASMD

Gastro-News, 2023
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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
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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
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A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results

Genetics in Medicine, 2022
Eugen Mengel, Roberto Giugliani, Michel Tchan   +2 more
exaly  

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
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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
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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
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Sustained Improvement of Interstitial Lung Disease Following Enzyme Replacement Therapy with Olipudase Alfa in Children and Adults with Chronic Acid Sphingomyelinase Deficiency

D98. TARGETING THE SCAR: MECHANISMS AND TREATMENTS FOR FIBROTIC LUNG DISEASE, 2022
Berger, K.I., Scarpa, M., Diaz, G.A., Giugliani, R., Lachmann, R., Wasserstein, M.P., Bonella, Francesco, Walsh, S.L.F., Wuyts, W.A., Jessel, A., Rawlings, A.M., Kumar, M.   +11 more
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eP280: Continued improvement in adults with acid sphingomyelinase deficiency after 2 years of olipudase alfa in the ASCEND placebo-controlled trial

Genetics in Medicine, 2022
Melissa Wasserstein, Antonio Barbato, Renata Gallagher, Roberto Giugliani, Norberto Guelbert, Julia Hennermann, Carla Hollak, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth Thurberg, Abhimanyu Yarramaneni, Andreea Rawlings, Yong Kim, Monica Kumar   +20 more
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Olipudase alfa

Reactions Weekly
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