Zobrazeno 1 - 10
of 795
pro vyhledávání: '"Immune tolerance induction ("'
Autor:
Zhengping Li, Jie Sun, Zekun Li, Zhenping Chen, Guoqing Liu, Wanru Yao, Xiaoling Cheng, Gang Li, Yingzi Zhen, Di Ai, Yaohan Zhou, Qianqian Mao, Man‐Chiu Poon, Runhui Wu
Publikováno v:
Pediatric Investigation, Vol 8, Iss 2, Pp 91-100 (2024)
ABSTRACT Importance It remained unclear that the efficacy comparison between low‐dose immune tolerance induction (LD‐ITI) incorporating immunosuppressants (IS) when severe hemophilia A (SHA) patients had inhibitor‐titer ≥200 Bethesda Units (B
Externí odkaz:
https://doaj.org/article/59e84e21e68e4af39c0dc1ebd74e8ec9
Autor:
Ángeles Palomo Bravo, Rosario Prieto Bonilla, Daniel Bardan Rebollar, Francisco José López‐Jaime, Ihosvany Fernández‐Bello
Publikováno v:
Clinical Case Reports, Vol 12, Iss 8, Pp n/a-n/a (2024)
Key Clinical Message Recommendations advise factor IX desensitization before immune tolerance induction in severe hemophilia B, supported by immunosuppression. A child with inhibitor and anaphylaxis to factor IX showed successful immunosuppression‐
Externí odkaz:
https://doaj.org/article/06613864f4aa4d3bb9558fdb0544fad6
Autor:
Nikita Chernyi, Darina Gavrilova, Mane Saruhanyan, Ezekiel S. Oloruntimehin, Alexander Karabelsky, Evgeny Bezsonov, Alexander Malogolovkin
Publikováno v:
Biomolecules, Vol 14, Iss 7, p 854 (2024)
One of the well-known X-linked genetic disorders is hemophilia, which could be hemophilia A as a result of a mutation in the F8 (factor VIII) gene or hemophilia B as a result of a mutation in the F9 (factor IX) gene, leading to insufficient levels of
Externí odkaz:
https://doaj.org/article/6c8e94f892a14917814386c7cf24cb85
Autor:
Ankit K. Desai, Garima Shrivastava, Christina L. Grant, Raymond Y. Wang, Trevor D. Burt, Priya S. Kishnani
Publikováno v:
Frontiers in Immunology, Vol 15 (2024)
IntroductionHigh sustained anti-rhGAA antibody titers (HSAT; ≥12,800) are directly linked to reduced efficacy of enzyme replacement therapy (ERT) and subsequent clinical deterioration in infantile-onset Pompe disease (IOPD). We have previously demo
Externí odkaz:
https://doaj.org/article/feaa2ff9617e4639b0282d4875ed2de3
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-8 (2023)
Abstract Background Haemophilia A is a bleeding disorder caused by deficiency of coagulation factor VIII (FVIII) which leads to severe and repeated bleedings. There is a need to understand the optimal treatment pathway for FVIII inhibitors with the u
Externí odkaz:
https://doaj.org/article/b852cc8a385c44a29c31e8f118c5f26f
Akademický článek
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Autor:
Qiushi Liang, Eva C. Vlaar, Fabio Catalano, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Wendy W.J. Unger, Ans T. van der Ploeg, W.W.M. Pim Pijnappel, Niek P. van Til
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 520-532 (2022)
Enzyme replacement therapy (ERT) is the current standard treatment for Pompe disease, a lysosomal storage disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). ERT has shown to be lifesaving in patients with classic infa
Externí odkaz:
https://doaj.org/article/833c3541c3c74a63aa8c8bff6fa7155d
Autor:
Vincenza Gragnaniello, Federica Deodato, Serena Gasperini, Maria Alice Donati, Clementina Canessa, Simona Fecarotta, Antonia Pascarella, Giuseppe Spadaro, Daniela Concolino, Alberto Burlina, Giancarlo Parenti, Pietro Strisciuglio, Agata Fiumara, Roberto Della Casa
Publikováno v:
Italian Journal of Pediatrics, Vol 48, Iss 1, Pp 1-14 (2022)
Abstract Background Classic infantile onset of Pompe disease (c-IOPD) leads to hypotonia and hypertrophic cardiomyopathy within the first days to weeks of life and, without treatment, patients die of cardiorespiratory failure in their first 1–2 yea
Externí odkaz:
https://doaj.org/article/61af1ccf535f4399a2639a84afdb50e6
Autor:
Jie Sun, Zekun Li, Gang Li, Kun Huang, Di Ai, Guoqing Liu, Wanru Yao, Xingjuan Xie, Hao Gu, Yingzi Zhen, Zhenping Chen, Runhui Wu
Publikováno v:
Research and Practice in Thrombosis and Haemostasis, Vol 6, Iss 7, Pp n/a-n/a (2022)
Abstract Background No studies evaluated the role of F8 mutations in outcomes for low‐dose immune tolerance induction (ITI) in people with severe hemophilia A (SHA) with high‐titer inhibitors. Objectives To explore the association between F8 muta
Externí odkaz:
https://doaj.org/article/c76cb85c86174975a8659eaaeae1c4fe
Autor:
Yuanhua Cai, Jocelyn A. Schroeder, Weiqing Jing, Cody Gurski, Calvin B. Williams, Shaoyuan Wang, Bonnie N. Dittel, Qizhen Shi
Publikováno v:
Frontiers in Immunology, Vol 13 (2022)
Multiple sclerosis (MS) is a chronic inflammatory autoimmune disease of the central nervous system with no cure yet. Here, we report genetic engineering of hematopoietic stem cells (HSCs) to express myelin oligodendrocyte glycoprotein (MOG), specific
Externí odkaz:
https://doaj.org/article/ec6a2fddd5f34326a50e945eeb517ef7