Givosiran

Givosiran (API)

Daim ntawv thov tshawb fawb:
Givosiran API yog ib qho hluavtaws me me cuam tshuam RNA (siRNA) tau kawm rau kev kho mob ntawm tus mob hepatic porphyria (AHP). Nws tshwj xeeb tsom rau covALAS 1noob (aminolevulinic acid synthase 1), uas yog koom nrog hauv heme biosynthesis txoj kev. Cov kws tshawb fawb siv Givosiran los tshawb xyuas RNA cuam tshuam (RNAi)-raws li kev kho mob, lub siab-targeted gene silencing, thiab kev hloov pauv ntawm cov txheej txheem metabolic koom nrog porphyria thiab cuam tshuam txog caj ces.

Muaj nuj nqi:
Givosiran ua haujlwm los ntawm kev txo cov kev qhia ntawmALAS 1nyob rau hauv hepatocytes, yog li txo qhov tsub zuj zuj ntawm cov tshuaj lom heme intermediates xws li ALA (aminolevulinic acid) thiab PBG (porphobilinogen). Qhov no pab tiv thaiv kev tawm tsam neurovisceral cuam tshuam nrog mob hepatic porphyria. Raws li API, Givosiran yog cov tshuaj siv tshuaj hauv RNAi-raws li kev kho mob tsim los muab kev tswj kav ntev ntawm AHP nrog kev tswj hwm subcutaneous.