Cov tshuaj menyuam ntsuag yog cov siv los kho cov kab mob tsawg. Lawv kuj hu ua "cov menyuam ntsuag". Zoo li yog tus kab mob no tsis tshua muaj, ces nyuaj rau nws.
Ib qho tsis sib haum xeeb tiag tiag ntawm kev ua haujlwm. Txawm hais tias qhov tsis tshua muaj, nyob rau hauv tag nrho muaj ntau tus neeg mob nrog xws li kev kuaj mob. Piv txwv li, nyob rau hauv cov teb chaws Europe muaj 30 lab ntawm lawv, uas yog, kwv yees li txhua txhua 15th tus neeg raug kev txom nyem los ntawm ib tug me nyuam ntsuag. Cov tshuaj tshwj xeeb yog npaj rau kev kho mob.
Niam txiv sau npe tshuaj
Cov tshuaj no feem ntau ua raws tib txoj kev tsim khoom thiab sau npe raws li cov tshuaj siv. Tab sis lawv tau txais qee cov txiaj ntsig uas txhawb cov tuam txhab lag luam los nqis peev hauv lawv txoj kev txhim kho.
tsoomfwv kev cuam tshuam los txhawb kev tsim khoom los ntawm ntau txoj hauv kev: kev so se thiab kev txhawb nqa, kev nkag tau yooj yim rau kev lag luam hauv tsev, kev txhawb nqa kev loj hlob, kev lag luam txuas ntxiv.
Leej twg assigns status
Txiv neej kev siv tshuaj yeeb tshuaj raug muabCov tshuaj los ntawm Pawg Neeg Saib Xyuas Kev Kho Mob Orphan, European Medicines Agency. Tom qab ntawd pom zoo los ntawm European Commission. Tsuas yog tom qab no yog daim ntawv pov thawj tso npe tawm.
Requirements
Yuav kom daim ntawv thov rau menyuam ntsuag raug pom zoo, nws yuav tsum ua raws li cov cai hauv qab no:
- Yuav tsum tau siv los kuaj xyuas, kho, lossis tiv thaiv kev ua rau lub neej lossis kab mob ntev.
- Tus kab mob yuav tsum tsis pub ntau tshaj 5 tus neeg ntawm 10,000 nyob rau hauv European Union lossis nws yuav tsum tsis zoo li kev muag khoom ntawm cov tshuaj yuav them rau cov nqi tsim nyog los tsim nws.
- Tam sim no tsis tsim nyog rau kev kuaj mob, kho lossis tiv thaiv tus kab mob lossis, yog tias tsim, cov tshuaj hauv nqe lus nug yuav tsum pab cov neeg mob uas muaj mob.
Tsab theem ntawm txoj cai
Hauv tebchaws Russia thaum kawg lub Peb Hlis 2010, daim nqi "Ntawm Kev Siv Tshuaj" tau pom zoo, uas muab rau lub xeev kev tswj hwm ntawm cov nqi tshuaj uas suav tias yog qhov tseem ceeb thiab tseem ceeb. Nyob rau tib lub sijhawm, tsis tau hais txog cov tshuaj hu ua menyuam ntsuag hauv qhov kawg version. Qhov kev hloov kho no ploj mus los ntawm txoj cai tom qab thawj qhov kev lees paub los ntawm Lub Xeev Duma.
Vim qhov no, ntau lub koom haum pab txhawb nqa tau koom ua kenyob rau hauv ib qho chaw nyob rau Thawj Tswj Hwm ntawm Russia. Lawv thov kom ua raws li kev cai lij choj ntawm cov tshuaj menyuam ntsuag thiab ua kom yooj yim rau tsoomfwv rau lawv cov ntshuam thiab sau npe hauv lub tebchaws.
Vim li ntawd, cov lus tshaj tawm raug tshaj tawm hauv lub vev xaib ntawm Ministry of He alth. Yog li, cov txheej txheem rau import cov tshuaj no tau yooj yim. Tam sim no tsis pub dhau 5 hnub.
Daim ntawv tso cai tso cai rau kev siv tus kheej yog muab tso rau hauv hluav taws xob hom nrog kos npe digital. Ua tsaug rau qhov no, cov neeg mob los ntawm thaj chaw deb yuav tsis tau mus xyuas lub peev, sawv hauv kab ntawm cov chaw ua haujlwm kom tau txais kev tso cai.
Cov kab mob menyuam ntsuag
Piv txwv cov kab mob menyuam ntsuag yog:
- YMucopolysaccharidosis. Cov kab mob tshwm sim nyob rau hauv daim ntawv hnyav, uas cuam tshuam txog cov kab mob metabolic. Hauv tebchaws Russia, qhov nruab nrab, kwv yees li 15 tus neeg yug los muaj tus kabmob no txhua xyoo.
- Hemophilia. Cov kab mob pathology nyob rau hauv uas qhov tsis txaus ntawm cov ntshav txhaws tau kuaj pom, uas ua rau cov txheej txheem qeeb. 8,000 tus neeg mob tau sau npe hauv Russia.
- mob mucous candidiasis. Tus neeg sawv cev ntawm tus kab mob yog fungus ntawm genus Candida. Feem ntau tshwm sim rau cov menyuam yaus hnub nyoog qis dua 18 xyoo.
Ntxiv rau, cov npe no suav nrog mucormycosis, zygomycosis, thymoma, malignant soft tissue sarcoma, cystic fibrosis, hyperprolactinemia, Rett syndrome, gastric ulcerative colitis, Majid syndrome, aniridia. Cov no tsis yog ib tug menyuam ntsuag pathologies - lawv cov npe ntev dua.
Lus npe tshuaj nrov
Cov tshuaj menyuam ntsuag nto moo tshaj plaws rau cov kab mob yog:
- "Soliris". Cov khoom xyaw nquag yog eculizumab. Cov tshuaj yog ib qho tshuaj tiv thaiv kab mob. Cov lus qhia rau kev siv - atypical hemolytic-uremic syndrome, nrog rau paroxysmal nocturnal hemoglobinuria. Contraindications muaj xws li nqa Neisseria meningitides, nquag kis kab mob, tsis muaj tshuaj tiv thaiv, mob siab heev thiab raum pathology.
- "Enplate". Lub ntsiab active ingredient yog romiplostim. Cov tshuaj no tau sau tseg rau idiopathic thrombocytopenic purpura.
- "Elapraza". Lub ntsiab tivthaiv yog idursulfase. Cov tshuaj no tau sau tseg rau mucopolysaccharidosis hom 2.
- "Trucklear". Cov khoom tseem ceeb hauv cov tshuaj yog bosentan. Nws yog siv rau pulmonary hypertension.
- "Enbrel". Cov tshuaj muaj etanercept. Nws raug sau tseg rau menyuam yaus mob caj dab nrog qhov pib tshwm sim.
- "Cia peb saib." Cov khoom xyaw nquag yog galsulfase. Cov cuab yeej siv rau mucopolysaccharidosis ntawm plaub yam.
- "Actemra". Muaj cov tocilizumab. Nws raug sau tseg rau menyuam yaus mob caj dab nrog qhov pib tshwm sim.
- "Revoleyd". Cov khoom xyaw nquag yog eltrombopag. Cov cuab yeej siv rau idiopathic thrombocytopenic purpura.
- "Aldazurim". Cov khoom tseem ceeb yog laronidase. Nws yog tshuaj rau mucopolysaccharidosis hom 1.
- "Ilaris". Cov khoom xyaw nquag yog canakinumab. Kev kho mob yog siv rau cov menyuam yaus mob caj dab nrog kev pib ua haujlwm.
- "Privigen". NwsCov khoom xyaw nquag yog tib neeg immunoglobulin. Txoj kev kho yog muab rau idiopathic thrombocytopenic purpura.
- "Txhob Cia Siab". Cov tshuaj nquag yog agalsidase alfa. Nws raug sau rau tus kab mob Fabry.
- "Exjade". Cov khoom xyaw nquag yog deferasirox. Cov tshuaj no yog siv rau qhov tsis tau qhia txog aplastic anemia.
- "Orfadin". Cov tshuaj nquag yog nitisinone. Nws raug sau tseg rau tyrosinemia.
- "Adempas". Muaj riociguat. Nws yog tshuaj rau pulmonary hypertension.
- "Zoo". Muaj macitentan. Nws yog siv rau pulmonary hypertension.
- "Revation". Muaj sildenafil. Tshuaj rau pulmonary hypertension.
- "Myozyme". Lub ntsiab tivthaiv yog alglucosidase alfa. Cov tshuaj no yog siv rau tus kab mob Pompe.
- "Vim". Lub ntsiab active ingredient yog miglustat. Nws raug sau tseg rau sphingolipidoses.
- "Ventavis". Muaj cov tshuaj iloprost. Tshuaj rau pulmonary hypertension.
- "Fabrazim". Muaj agalsidase beta. Nws raug sau tseg rau sphingolipidoses.
- "Volibris". Lub active compound yog ambisentan. Tshuaj rau pulmonary hypertension.
- "Ilomedin". Cov khoom xyaw nquag yog iloprost. Cov tshuaj no yog siv rau pulmonary hypertension.
Daim ntawv teev cov tshuaj menyuam ntsuag no suav hais tias yog qhov nrov tshaj plaws ntawm cov kws kho mob.
YContraindications
Tshuaj yog txwv tsis pub noj thaum twgnce susceptibility rau lub active compound ntawm cov tshuaj, raws li zoo raws li nyob rau hauv cov ntaub ntawv loj ntawm daim siab thiab raum pathologies. Cov me nyuam tsuas yog tom qab tau tso cai los ntawm tus kws kho mob xwb.
Zoo kawg
Yog li, daim ntawv teev cov tshuaj menyuam ntsuag yog cov npe tshuaj uas tau sau tseg rau cov kab mob los ntawm pawg tib lub npe. Xws li pathologies suav hais tias tsis tshua muaj.
Rau kev txhim kho cov tshuaj menyuam ntsuag, muaj kev txhawb siab rau cov tuam txhab. Kev sau npe yog ua tiav ib yam li rau lwm yam tshuaj, tab sis nrog kev pom zoo ntxiv los ntawm cov koom haum cuam tshuam.
Rau cov neeg mob, yuav tsum nco ntsoov tias tsuas yog kws kho mob yuav tsum tau sau tshuaj. Kev yuav cov tshuaj rau tus kheej tsis pom zoo, vim nws yuav tsis tuaj yeem ua rau qhov xav tau, tab sis tsuas yog ua rau tus kab mob hnyav dua.
