API Oligonucleotide
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Mbelata sodium
Inclisiran sodium API (Active Pharmaceutical Ingredient) ka a na-amụ nke ọma na nnyonye anya RNA (RNAi) na ọgwụgwọ ọrịa obi. Dị ka siRNA nwere eriri okpukpu abụọ na-ezubere iche maka mkpụrụ ndụ PCSK9, a na-eji ya na nyocha izizi yana nyocha ụlọ ọgwụ iji nyochaa usoro ịgbachi nkịtị na-eme ogologo oge maka iweda LDL-C (cholesterol lipoprotein dị ala). Ọ na-arụkwa ọrụ dị ka onyinye ihe atụ maka nyocha sistemu nnyefe siRNA, nkwụsi ike, yana ọgwụgwọ RNA lekwasịrị anya imeju.
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Donidalorsen
Donidalorsen API bụ antisense oligonucleotide (ASO) na-enyocha nyocha maka ọgwụgwọ angioedema eketa (HAE) na ọnọdụ mkpali metụtara ya. A na-amụ ya na usoro ọgwụgwọ ezubere iche maka RNA, na-achọ ibelata okwu nkeplasma prekallikrein(KLKB1 mRNA). Ndị na-eme nchọpụta na-eji Donidalorsen chọpụta usoro ịgbachi nkịtị n'ime mkpụrụ ndụ ihe nketa, ọgwụ ọgwụ na-adabere na dose, na njikwa ogologo oge nke mbufụt mgbasa ozi bradykinin.
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Fitusiran
Fitusiran API bụ RNA (siRNA) nke na-etinye aka na-arụ ọrụ nke a nyochara nke ọma na ngalaba hemophilia na ọrịa coagulation. Ọ lekwasịrị anyaantithrombin (AT ma ọ bụ SERPINC1)mkpụrụ ndụ ihe nketa na imeju iji belata mmepụta antithrombin. Ndị na-eme nchọpụta na-eji Fitusiran nyochaa usoro nnyonye anya RNA (RNAi), ịgbachi nkịtị n'ime imeju, yana usoro ọgwụgwọ ọhụụ maka ịhazigharị coagulation na ndị ọrịa hemophilia A na B, yana ma ọ bụ na-enweghị ihe mgbochi.
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Givosiran
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.
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Plozasiran
Plozasiran API bụ obere RNA (siRNA) nke na-etinye aka na ya emebere maka ọgwụgwọ hypertriglyceridemia na ọrịa obi na ọrịa metabolic metụtara ya. Ọ lekwasịrị anyaAPOC3mkpụrụ ndụ ihe nketa, nke na-etinye koodu apolipoprotein C-III, bụ isi na-achịkwa metabolism triglyceride. N'ime nyocha, a na-eji Plozasiran mụọ usoro mgbada lipid dabere na RNAi, nkọwapụta mkpụrụ ndụ ihe nketa, yana ọgwụgwọ ogologo oge maka ọnọdụ dị ka ọrịa chylomicronemia nke ezinụlọ (FCS) na dyslipidemia agwakọta.
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Zilebesiran
Zilebesiran API bụ nyocha obere RNA (siRNA) na-etinye aka na nyocha nke emepụtara maka ọgwụgwọ ọbara mgbali elu. Ọ lekwasịrị anyaAGTmkpụrụ ndụ ihe nketa, nke na-etinye koodu angiotensinogen - akụkụ bụ isi nke sistemu renin-angiotensin-aldosterone (RAAS). Na nyocha, a na-eji Zilebesiran amụ usoro ịgbachi nkịtị maka mkpụrụ ndụ ọbara mgbali elu ogologo oge, teknụzụ nnyefe RNAi, yana ọrụ sara mbara nke ụzọ RAAS na ọrịa obi na akụrụ.