Givosiran

Givosiran (API)

Ngwa nyocha:
Givosiran API bụ RNA (siRNA) nke na-etinye aka na ya nke a na-amụ maka ọgwụgwọ nke nnukwu imeju porphyria (AHP). Ọ kpọmkwem lekwasịrị anya naALAS1gene (aminolevulinic acid synthase 1), nke na-etinye aka na heme biosynthesis ụzọ. Ndị na-eme nchọpụta na-eji Givosiran nyochaa nnyonye anya RNA (RNAi), usoro ọgwụgwọ na-adabere na imeju, na imeju nke ụzọ metabolic na-etinye aka na porphyria na ọrịa mkpụrụ ndụ ihe nketa metụtara ya.

Ọrụ:
Givosiran na-arụ ọrụ site na ibelata okwu nkeALAS1na hepatocytes, si otú ahụ na-ebelata mkpokọta nke heme intermediates na-egbu egbu dị ka ALA (aminolevulinic acid) na PBG (porphobilinogen). Nke a na-enyere aka igbochi mwakpo neurovisceral jikọtara ya na nnukwu hepatic porphyria. Dị ka API, Givosiran bụ akụrụngwa ọgwụ na-arụ ọrụ na ọgwụgwọ dabere na RNAi emebere iji nye njikwa ogologo oge nke AHP na nchịkwa subcutaneous.