Zobrazeno 1 - 10
of 1 464
pro vyhledávání: '"sebelipase alfa"'
Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.
Autor:
de Castro, María José1,2 (AUTHOR), Jones, Simon A3,4 (AUTHOR) simonallanjones@gmail.com, de las Heras, Javier5 (AUTHOR), Sánchez-Pintos, Paula1,2 (AUTHOR), Couce, María L1,2 (AUTHOR), Colón, Cristóbal1,2 (AUTHOR), Crujeiras, Pablo1,2 (AUTHOR), Unceta, María5 (AUTHOR), Church, Heather3 (AUTHOR), Brammeier, Kathryn3 (AUTHOR), Yee, Wu Hoi3 (AUTHOR), Cooper, James3 (AUTHOR), López de Frutos, Laura6 (AUTHOR), Serrano-Gonzalo, Irene6 (AUTHOR), Camba, María José1,2 (AUTHOR), White, Fiona J.3 (AUTHOR), Holmes, Victoria3 (AUTHOR), Ghosh, Arunabha3,4 (AUTHOR)
Publikováno v:
Orphanet Journal of Rare Diseases. 6/25/2024, Vol. 19 Issue 1, p1-9. 9p.
Autor:
María José de Castro, Simon A Jones, Javier de las Heras, Paula Sánchez-Pintos, María L Couce, Cristóbal Colón, Pablo Crujeiras, María Unceta, Heather Church, Kathryn Brammeier, Wu Hoi Yee, James Cooper, Laura López de Frutos, Irene Serrano-Gonzalo, María José Camba, Fiona J. White, Victoria Holmes, Arunabha Ghosh
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-9 (2024)
Abstract Background Sebelipase alfa (Kanuma®) is approved for patients with Wolman disease (WD) at a dosage of 3–5 mg/kg once weekly. Survival rates in the second of two clinical trials was greater, despite recruiting more severely ill patients, p
Externí odkaz:
https://doaj.org/article/b5c38565cdd44d16807759d4ee950516
Akademický článek
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Publikováno v:
In Journal of Hepatology March 2022 76(3):577-587
Autor:
Michelle F. Huffaker, Anne Y. Liu, Gregory M. Enns, Suresh Vijay, Antonio J. Amor, N. Franklin Adkinson Jr
Publikováno v:
JIMD Reports, Vol 49, Iss 1, Pp 30-36 (2019)
Abstract Allergic immune‐mediated hypersensitivity reactions are known potential complications of enzyme replacement therapy. Sebelipase alfa, recombinant lysosomal acid lipase (LAL), is a potentially life‐altering treatment for patients with LAL
Externí odkaz:
https://doaj.org/article/125628128ec746fc87d513943caf75e0
Autor:
Vijay, Suresh1 (AUTHOR) suresh.vijay1@nhs.net, Brassier, Anais2 (AUTHOR), Ghosh, Arunabha3 (AUTHOR), Fecarotta, Simona4 (AUTHOR), Abel, Florian5 (AUTHOR), Marulkar, Sachin5 (AUTHOR), Jones, Simon A.3 (AUTHOR)
Publikováno v:
Orphanet Journal of Rare Diseases. 3/2/2021, Vol. 16 Issue 1, p1-16. 16p.
Autor:
Tanguy Demaret, Florence Lacaille, Camille Wicker, Jean-Baptiste Arnoux, Juliette Bouchereau, Claire Belloche, Cyril Gitiaux, David Grevent, Christine Broissand, Dalila Adjaoud, Marie-Thérèse Abi Warde, Dominique Plantaz, Soumeya Bekri, Pascale de Lonlay, Anaïs Brassier
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-9 (2021)
Abstract Background Wolman disease (WD), the rapidly progressive phenotype of lysosomal acid lipase (LAL) deficiency, presents in neonates with failure to thrive and hepatosplenomegaly, and leads to multi-organ failure and death before 12 months of a
Externí odkaz:
https://doaj.org/article/a789869fae7343719234174d01ea84b9
Autor:
Malinová, Vĕra1 (AUTHOR) vera.malinova@vfn.cz, Balwani, Manisha2 (AUTHOR), Sharma, Reena3 (AUTHOR), Arnoux, Jean‐Baptiste4 (AUTHOR), Kane, John5 (AUTHOR), Whitley, Chester B.6 (AUTHOR), Marulkar, Sachin7 (AUTHOR), Abel, Florian7 (AUTHOR)
Publikováno v:
Liver International. Sep2020, Vol. 40 Issue 9, p2203-2214. 12p. 2 Diagrams, 3 Charts, 3 Graphs.
Akademický článek
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Publikováno v:
In Journal of Clinical Lipidology May-June 2018 12(3):604-614