Zobrazeno 1 - 10
of 150
pro vyhledávání: '"givosiran"'
Autor:
Eliane Sardh, Manisha Balwani, David C. Rees, Karl E. Anderson, Gang Jia, Marianne T. Sweetser, Bruce Wang
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-14 (2024)
Abstract Background Acute hepatic porphyria is a group of multisystem disorders of which acute intermittent porphyria is the most common subtype. Givosiran, a subcutaneously administered RNA interference therapeutic targeting liver ALAS mRNA, is appr
Externí odkaz:
https://doaj.org/article/759857acf82a4a949b1e43939d2fdb36
Publikováno v:
JIMD Reports, Vol 65, Iss 4, Pp 262-271 (2024)
Abstract Givosiran is a subcutaneously administered, liver‐targeted RNA interference (RNAi) therapeutic that has been approved for treating acute hepatic porphyria (AHP). Elevation in plasma homocysteine (hyperhomocysteinemia) has been reported in
Externí odkaz:
https://doaj.org/article/bbd72c333fd44ced838ecb21916b0238
Publikováno v:
Journal of the Formosan Medical Association, Vol 123, Iss 6, Pp 679-686 (2024)
Background/purpose: Acute hepatic porphyrias (AHP) are rare genetic disorders associated with acute neurovisceral attacks and chronic symptoms. This analysis was conducted to examine the long-term efficacy and safety of givosiran in Taiwanese partici
Externí odkaz:
https://doaj.org/article/6b9f61050b21447189c634e505d495e5
Autor:
Claudio Carmine Guida, Maria Nardella, Aurora del Mar YS Perez, Maria Savino, Gaetano Ferrara, Francesco Napolitano, Annalisa Crisetti, Francesco Aucella, Filippo Aucella
Publikováno v:
Molecular Genetics and Metabolism Reports, Vol 40, Iss , Pp 101111- (2024)
Acute hepatic porphyrias (AHPs) are a family of rare, autosomal, dominantly inherited conditions characterized by abnormalities in the production of heme. Advances in molecular engineering have provided new therapeutic possibilities for modifying the
Externí odkaz:
https://doaj.org/article/9d9052a1050d40ac9f42682858fc1336
Autor:
Isabelle Redonnet-Vernhet, Patrick Mercié, Louis Lebreton, Jean-Marc Blouin, Didier Bronnimann, Samir Mesli, Claire Guibet, Emmanuel Ribeiro, Noémie Gensous, Pierre Duffau, Laurent Gouya, Emmanuel Richard
Publikováno v:
Molecular Genetics and Metabolism Reports, Vol 39, Iss , Pp 101076- (2024)
Acute hepatic porphyrias are inherited metabolic disorders of heme biosynthesis characterized by the accumulation of toxic intermediate metabolites responsible for disabling acute neurovisceral attacks. Givosiran is a newly approved siRNA-based treat
Externí odkaz:
https://doaj.org/article/3f6cf5bab6a04cf087bfd971ca90b02d
Autor:
Paulo Victor Sgobbi Souza, Gliciane Afonso, Wladimir Bocca Vieira de Rezende Pinto, Paulo de Lima Serrano, Bruno de Mattos Lombardi Badia, Igor Braga Farias, Ana Carolina dos Santos Jorge, Roberta Ismael Lacerda Machado, Icaro França Navarro Pinto, Glenda Barbosa Barros, Helvia Bertoldo de Oliveira, Samia Rogatis Calil, Cibele Franz, Acary Souza Bulle Oliveira
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-11 (2023)
Abstract Background Porphyrias are a rare group of disease due to inherited defects of heme synthesis with important systemic manifestations and great burden of disease for patients and families due to the exceptional course of disease with disabling
Externí odkaz:
https://doaj.org/article/e991abfc8f334db599cba5bf626ab962
Publikováno v:
Pharmacy, Vol 12, Iss 2, p 58 (2024)
Small interfering RNA (siRNA)-based medications offer the ability to target previously undruggable targets and have now received FDA approval in five instances for orphan or uncommon diseases. The current siRNA “-sirans” are directed towards hepa
Externí odkaz:
https://doaj.org/article/303799104901456da75f2b62acf399b1
Akademický článek
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Akademický článek
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Autor:
Bruce Wang, Paolo Ventura, Kei-ichiro Takase, Manish Thapar, David Cassiman, Ilja Kubisch, Shangbin Liu, Marianne T. Sweetser, Manisha Balwani
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-12 (2022)
Abstract Background Acute hepatic porphyria (AHP) is a family of four rare genetic diseases, each involving deficiency in a hepatic heme biosynthetic enzyme. Resultant overproduction of the neurotoxic intermediates δ-aminolevulinic acid (ALA) and po
Externí odkaz:
https://doaj.org/article/126628ee581049f09f0b982642040408