Yini ushayele imithi ezibizayo kakhulu emhlabeni?

Izimali izinkampani ezithile ezenza imithi agingqa ezingaphezu kwezigidi eziyinkulungwane. Lokhu kungenxa yokuthi nokuhambisa imithi ezibizayo, ekhiqizwa ngamaqoqo amancane. Ngokuvamile lokhu imithi, ugweme pathologies ezingavamile. Abanye ochwepheshe bakholelwa ukuthi izindleko zemithi ethile siphezulu kakhulu ngenxa yokuthi akukho mncintiswano. Uhlu imikhiqizo efana kuyinto ngempela ebanzi, phakathi kwawo eqolo emhlabeni wezokwelapha izobiza abangaphezu kwesigidi euro.

Umuthi Aldurazyme

Lesi sidakamizwa yakhelwe aphathe Hurler syndrome, esizibonakalisa ebuntwaneni. Uphethwe ekuguleni abantu kancane kuka angamakhulu ayisithupha emhlabeni wonke. Nokugula kuvimbela ukuthuthukiswa ngokomzimba nangokokuziphatha kwezingane ezineminyaka emine ubudala.

Izindleko zalesi luhlobo cishe amadola ayizinkulungwane ezingamakhulu amabili.

Izidakamizwa "Cerezyme"

At izindleko lokhu izidakamizwa izobiza mayelana esilinganayo njengoba Aldurazyme. "Cerezyme" - kuyafana umuthi ezibizayo kakhulu emhlabeni. It ezinqunyiwe Gaucher isifo. Ukuze lesi sifo libhekene ekuqongeleleni okunamafutha ezigcine emaphashini, isibindi, ubuchopho kanye umnkantsha. Lolu hlobo lantambama ukukhuluphala umsebenzi eziyinkimbinkimbi kwamaphaphu nezinso. Imithi ubuyisela enzyme ungaphakathi Gaucher isifo engekho nhlobo.

Emhlabeni wonke, abantu yilesi sifo abantu abangaba yizinkulungwane eziyisithupha.

I Myozyme izidakamizwa

Kunezinhlobo ezingaba ngu-emakhulwini izidakamizwa, okwabangela inhlekelele engaphezu kwenkulungwane euro isitsha ngasinye. Zonke lezi izidakamizwa basonta isigaba "imithi kakhulu eqolo emhlabeni." Yini igama lomunye izidakamizwa kuleli qembu? Lo muthi Myozyme, ukwelapha Pompe syndrome. A zokugula libhekene nokuntuleka-alpha-glucosidase. Lokhu kuholela ekubhujisweni imisipha futhi waba ukwanda okukhulu kwezincwadi ngevolumu inhliziyo, ukuthuthukisa zokugula zokuphefumula. Ngokuvamile, ukugula kutholwa ebuntwaneni.

Ukusetshenziswa kwezidakamizwa ungayeka izinqubo futhi usiza izingane ziqala ukushaya futhi sakwazi ukuphefumula ngokuzimela. Lokho okwakudingeka ukuze kukhokhwe ejenti ukwelashwa izobiza ayizinkulungwane eziyikhulu euro ingane. Uma lesi sifo sitholakale umuntu isikhulile, ke ukwelashwa kuzothatha abangaphezu kwamakhulu ayizinkulungwane ezintathu.

kusho Fabrazyme

Omunye imithi ebiza kakhulu emhlabeni - ke kuyindlela yokuthola Fabrazyme. Izindleko ukwelashwa izidakamizwa izobiza mayelana izinkulungwane ezingamakhulu amathathu amadola.

Umuthi zenzelwe ekwelapheni Fabry isifo. Kubangela a ukuncinza, inhliziyo lakhulisiwe, izinso izinkinga. Emhlabeni wonke, abantu abaphethwe yilesi sifo, hhayi ningi than abantu abayizinkulungwane ezintathu.

I Arcalyst izidakamizwa

Abanye bakholelwa ukuthi kuyabiza kakhulu emhlabeni of imithi - umkhiqizi izidakamizwa Regeneron ngokuthi Arcalyst, ukwelapha Syndrome Muckle-Wales. Pathology is ezilokhu zivela imfiva ngakanye, ukuqaqamba kwamalunga omzimba, izinso. Izifo kuthinta abantu abangaba yizigidi ezimbili emhlabeni wonke.

Izindleko zomuthi imayelana ezimbili eziyizinkulungwane eziyikhulu namashumi amabili amadola.

I Soliris izidakamizwa

Esikhathini esithile esidlule, umuthi ezibizayo kakhulu emhlabeni - Soliris. Intengo zomuthi cishe kwesigamu ayisigidi. Kusho ekwelapheni ukuphazamiseka lapho izivikeli mzimba amangqamuzana abomvu egazi. Lokhu imithi esithathwayo acishe abe yizinkulungwane ezinhlanu.

Imithi Elaprase Naglazyme

Enye izidakamizwa kusukela isigaba imithi ebiza kakhulu emhlabeni - kuyinto Elaprase izidakamizwa. Izindleko Its ayizinkulungwane ezingamakhulu amane amadola. Kusho kuboniswe ngoba Hunter syndrome. Ukuze pathologies libhekene i ukwanda kolimi, ukulimala kobuchopho futhi ekhaleni eshelelayo. Emhlabeni wonke, lokhu izidakamizwa kudinga abantu kungathi izinkulungwane ezimbili.

Cishe izindleko ezifanayo Naglazyme. It ezinqunyiwe ukwelapha Maroto-Lamy. Sifo sithinta ngaphezulu kwenkulungwane abantu emhlabeni wonke. Ukuze lesi sifo libhekene izindebe awugqinsi, onesiqu esifushane futhi ikhanda elikhulu. Kakade kusukela kwaneminyaka iziguli nanhlanu ngesihlalo sabakhubazekile.

Cinryze

Lesi sidakamizwa - umuthi ezibizayo kakhulu emhlabeni futhi zamaRandi angaphezu kuka enamandla eyizinkulungwane ezingamakhulu amathathu. Umuthi yakhelwe aphathe lezinceku angioedema hlobo. Kulesi ukugula wabonakaliswa kahle amasosha omzimba, kubangela ukuvuvukala ezinzima sezindebe, komgodi ongaphakathi kwamathumbu.

Umuthi eqolo

Ngakho yini imithi ezibizayo kakhulu emhlabeni? Kungakanani Kubiza? izinkampani ezimbili zakwamanye amazwe baye bahlakulela imithi ezibizayo kakhulu emhlabeni. Glybera ibizwa ngokuthi. Izindleko zokwelapha lesi sakhiwo kuyoba ngaphezu bathole izigidi zamaRandi.

Emisha yezakhi zofuzo imithi kuhloswe ukuba aphathe ezingavamile isifo esidluliselwa ngofuzo - Esilula lipase ukuntula. Enzyme ulawula ezingeni lipids emzimbeni. Ngenxa isakhi sofuzo kuguquka enzyme ibizwa hhayi ezivezwa umzimba, ngoba ingozi eliphezulu occlusion semithambo.

Omunye umfuma izidakamizwa izobiza ezingaphezu kwamashumi amahlanu amawaka euro, futhi izifundo egcwele nokwelashwa kwezifo kungakephuzi kuyoba abangaphezu kwesigidi. iziguli ocatshangelwayo nale sifo, kunabantu kungathi izingalo ezingamakhulu amabili e-Europe, ngakho ithuluzi akunakwenzeka ukuba uneminye umthelela kwesabelomali izikhungo zezempilo.

Naphezu amanani aphezulu, ithuluzi uyakwazi ukwelapha ngokuphelele egunjini lokuhlolwa kwezifo. Ngokwe-developers, imithi oyinikiwe ne eshibhile amaningi yokwelapha enzyme esikhundleni.

Umuthi Glybera isakhi sofuzo isikhungo sesine emhlabeni. Okokuqala okubili ezakhiwa ososayensi American futhi eChina. Bobabili amathuluzi zenzelwe wokwelapha isifo somdlavuza, okungukuthi, umdlavuza womlomo wesibeletho kanye izimila ikhanda. izindlela Okwesithathu eRussia. Leli thuluzi ngokuthi "Neovaskulgen", okuhloswe for ekwelapheni aphansi esitho ischemia. Inkambo yokwelashwa nale imithi izobiza ruble mayelana ezinkulungwaneni ezingamakhulu amathathu.

ejensi Biotechnological bakholelwa ukuthi imithi ebiza kakhulu emhlabeni, izithombe zazo zingatholakala esihlokweni ufaneleke uqobo. Empeleni, kuze kube kufika le mithi, abantu abaningi abaphethwe pathologies sina, asikwazanga ukuqeda izifo zabo. Izinkampani ezithaka amakhambi kukhona inzuzo ukuthengisa le mithi, kusukela izikhangiso zabo akudingeki ukuchitha imali. Futhi le mithi cishe akukho umncintiswano, okuyinto eyenza abakhiqizi ngokwabo ukunquma izindleko zomuthi futhi ukutholakala kwayo.

Esikhathini umshwalense United States izinkampani buyisela izindleko izidakamizwa ezibizayo iziguli, futhi abanye abakhiqizi ukunikela khulula imithi abaswele.

Ochwepheshe emhlabeni wonke ukuze bathethelele zibiza kwemithi emisha. Ngokusho kwabo, ukusetshenziswa imithi enjalo zivumele iziguli eziningi kancane, futhi kwezinye izimo alulame ngokuphelele ukususa syndromes ezingelapheki. Ochwepheshe bakholelwa ukuthi intengo lezi izidakamizwa yehlile, umncintiswano kuyadingeka. abantu abambalwa kuphela zindla labo baye bathola izifo ezimbi, ngisho esemncane, cishe ngeke ukwazi ukuthenga izidakamizwa, kubiza izinkulungwane. Ezimweni eziningi, abantu abanayo ithuba ukuthenga imithi enjalo ezibizayo.