[7] Guo S, Booten SL, Watt A, Alvarado L, Freier SM, Teckman JH, McCaleb ML, Monia BP. Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease. Rare Dis. 2014;2:e28511. pii: 2014RAREDIS066R. [8] ...
参考资料: New form of gene editing efficient for treatment of AATD, liver and lung disease (medicalxpress.com) 版权说明:本文来自医伴旅内容团队,欢迎个人转发至朋友圈,谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在...
Mandorfer M, Choudhury G, Griffiths W, Trautwein C, Loomba R, Schluep T, Chang T, Yi M, Given BD, Hamilton JC, San Martin J, Teckman JH.Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency.N Engl J Med . 2022 Jun 25....
AATD UpdateAlpha-1 antitrypsin (AAT) deficiency is a genetic disorder clinically characterized by early-onset emphysema and liver disease. First recognized in 1963, AAT deficiency is commonly regarded as a disease of Caucasians of northern European descent. 1 The underlyingJK Stoller...
X Mieli-Vergani G, Doherty DG, Donaldson PT, et al. HLA status in children with alpha-i antitrypsin deficiency (AATd) (PiZ) and chronic liver disease (CLD). Pediatr Res 1987;22:98.Mieli-Vergani G, Doherty DG, Donaldson PT, et al. HLA status in children with alpha-1-antitrypsin ...
Vertex Pharma was lower after the drugmaker said it would not pursue VX-864, a drug to treat alpha-1 antitrypsin deficiency, into late-stage development.
SEQUOIA (NCT03945292) is a placebo-controlled, adaptive design Phase 2/3 study to evaluate the safety, efficacy, and tolerability of ARO-AAT administered subcutaneously to patients with AATD associated liver disease. Arrowhead Pharmaceuticals Doses First Patient in SEQUOIA Phase 2/3 Study of ARO-AA...
Liver Disease in Alpha-1 Antitrypsin Deficiency: Current Understanding and Future Therapy Alpha-1 antitrypsin Deficiency (AATD) is a common, but under recognized metabolic genetic disease. Although many mutations in the alpha-1 antitrypsin (AAT)... Teckman,H Jeffrey - 《Copd Journal of Chronic ...
Alpha-1 antitrypsin deficiency (AATD) is a hereditary condition associated with a higher risk of developing lung disease, in the form of emphysema, and liver disorders, depending on the allelic variants expressed [1]. Currently, the measurement of alpha-1 antitrypsin levels is recommended in all...
X Mieli-Vergani G, Doherty DG, Donaldson PT, et al. HLA status in children with alpha-i antitrypsin deficiency (AATd) (PiZ) and chronic liver disease (CLD). Pediatr Res 1987;22:98.Mieli-Vergani G, Doherty DG, Donaldson PT, et al. HLA status in children with alpha-1-antitrypsin ...