Givosiran

Givosiran (API)

Tšebeliso ea Lipatlisiso:
Givosiran API ke RNA e nyane e sitisang e nyane (siRNA) e ithutoang bakeng sa kalafo ea acute hepatic porphyria (AHP). E shebane ka ho khetheha leALAS1gene (aminolevulinic acid synthase 1), e amehang tseleng ea heme biosynthesis. Bafuputsi ba sebelisa Givosiran ho batlisisa mekhoa ea phekolo e thehiloeng ho RNA (RNAi), ho khutsisa liphatsa tsa lefutso tse lebisitsoeng ho sebete, le ho feto-fetoha ha mekhoa ea metabolic e amehang ho porphyria le mathata a amanang le lefutso.

Mosebetsi:
Givosiran mesebetsi ka ho fokotsa polelo eaALAS1ka li-hepatocyte, ka hona ho fokotsa ho bokellana ha li-heme tse chefo tse kang ALA (aminolevulinic acid) le PBG (porphobilinogen). Sena se thusa ho thibela litlhaselo tsa neurovisceral tse amanang le acute hepatic porphyria. Joaloka API, Givosiran ke karolo e sebetsang ea meriana ea phekolo e thehiloeng ho RNAi e etselitsoeng ho fana ka taolo ea nako e telele ea AHP ka tsamaiso ea subcutaneous.