Givosiran

Givosiran (API)

Codsiga Cilmi-baarista:
Givosiran API waa RNA (siRNA) faragelin yar oo synthetic ah oo loo bartay daaweynta cagaarshow ba'an (AHP). Waxay si gaar ah u beegsanaysaaALAS1hiddo-wadaha (aminolevulinic acid synthase 1), kaas oo ku lug leh dariiqa biosynthesis ee heme. Cilmi-baadhayaashu waxay isticmaalaan Givosiran si ay u baaraan faragelinta RNA (RNAi) daaweynta ku salaysan, aamusnaanta hiddo-wadaha beerka, iyo habeynta hababka dheef-shiid kiimikaadka ee ku lug leh porphyria iyo cilladaha hiddaha ee la xiriira.

Shaqada:
Givosiran wuxuu u shaqeeyaa isagoo yareynaya muujintaALAS1Hepatocytes, taas oo hoos u dhigaysa ururinta heme dhexe ee sunta ah sida ALA (aminolevulinic acid) iyo PBG (porphobilinogen). Tani waxay kaa caawinaysaa ka hortagga weerarrada neurovisceral ee la xidhiidha porphyria cagaarshow ba'an. API ahaan, Givosiran waa qayb ka mid ah dawooyinka firfircoon ee daweynta RNAi-ku-salaysan ee loogu talagalay in lagu bixiyo xakamaynta muddada-dheer ee AHP oo leh maamulka maqaarka hoostiisa.