Donidalorsen (API)
Daim ntawv thov tshawb fawb:
Donidalorsen API yog ib qho antisense oligonucleotide (ASO) nyob rau hauv kev tshawb nrhiav rau kev kho mob ntawm hereditary angioedema (HAE) thiab lwm yam mob. Nws yog kawm nyob rau hauv cov ntsiab lus ntawm RNA-targeted kev kho mob, aiming kom txo tau cov kev qhia ntawmntshav plasma prekallikrein(KLKB1 mRNA). Cov kws tshawb fawb siv Donidalorsen los tshawb nrhiav cov tshuaj gene silencing mechanisms, koob tshuaj raws li pharmacokinetics, thiab kev tswj mus sij hawm ntev ntawm bradykinin-mediated o.
Muaj nuj nqi:
Donidalorsen ua haujlwm los ntawm kev xaiv khi rauKLKB1mRNA, txo cov ntshav plasma prekallikrein - ib qho tseem ceeb enzyme nyob rau hauv lub kallikrein-kinin system lub luag hauj lwm rau ua o thiab o nyob rau hauv HAE. Los ntawm kev txo qis cov qib kallikrein, Donidalorsen pab tiv thaiv HAE tawm tsam thiab txo cov kab mob. Raws li API, nws ua haujlwm tseem ceeb hauv kev txhim kho kev ua haujlwm ntev, kev kho mob subcutaneously rau HAE.