Yintoni na ukuba uthi iyeza kuyabiza kakhulu ehlabathini?

Ngongeniselo iinkampani ezithile zoxubo waqengqela angaphezu kwezigidi. Oku kungenxa yokuba ke beninikela iziyobisi eninzi, ekuveliseni ngokwamaqela amancinane. Ngokuqhelekileyo oku amayeza, ukuphepha ezimbi ezinqabileyo. Ezinye iingcali zikholelwa ukuba ixabiso amanye amayeza liphezulu kakhulu ngenxa yokuba akukho khuphiswano. Uluhlu iimveliso enjalo ebanzi kakhulu, phakathi kwabo ezibiza kakhulu kwihlabathi yamayeza uya kubiza ngaphezu kwesigidi euro.

Ichiza Aldurazyme

Eli chiza yenzelwe ukunyanga Hurler syndrome, esizibonakalisa ebuntwaneni. Uphethwe sisigulo abantu kancinane amathandathu, anamakhulu ehlabathini lonke. Ukugula abangaphangeliyo uphuhliso emzimbeni yokuziphatha kwabantwana iminyaka ezineminyaka emine ubudala.

Indleko yale sibonelelo malunga angamakhulu amabini amawaka eerandi.

Drug "Cerezyme"

Xa iindleko eli chiza iya kuxabisa malunga elifanayo naleyo Aldurazyme. "Cerezyme" - iyafana nje amayeza kuyabiza kakhulu ehlabathini. It omiselwe isifo Gaucher. Kuba sifo luphawulwa beqokelela iidiphozithi ngamanqatha emiphungeni, isibindi, ingqondo kunye ithambo umongo. Olu hlobo ebunayo ukutyeba umsebenzi elinzima emiphungeni kunye nezintso. Amayeza ithatha enzyme na izifo Gaucher engekho ngokupheleleyo.

Ehlabathini lonke, abantu nale syndrome malunga namawaka amathandathu ngabantu.

Le Myozyme kweziyobisi

Kukho malunga amakhulu iziyobisi, endiwazuze ngaphezu kwewaka euro igutyana nganye. Onke la machiza kuba kudidi "kakhulu amayeza eqolo ehlabathini." Yintoni igama kwelinye ichiza kweli qela? Olu nyango Myozyme, kuba unyango lwe Pompe syndrome. A pathology lubonakala ngokuba ukuswela alpha-glucosidase. Oku kukhokelela ukutshatyalaliswa izihlunu kunye nokwanda okukhulu umthamo wentliziyo, ukuphuhlisa kwizifo sokuphefumla. Amaninzi, esi sifo ibonakele ebuntwaneni.

Ukusetyenziswa kweziyobisi enokuthintela iinkqubo yaye inceda abantwana baqale ukuphefumla ngokuzimeleyo. Iindleko arhente imithi iya kuxabisa ikhulu lamawaka euro umntwana. Ukuba isifo eso nasebudaleni, ngoko unyango uya ngaphezu kwamakhulu amathathu amawaka.

kuthetha Fabrazyme

Unelinye iyeza kuyabiza kakhulu ehlabathini - oko uThixo kukusekeleza Fabrazyme. Iindleko zonyango kweziyobisi iya kuxabisa malunga namakhulu amathathu amawaka eerandi.

Ichiza iphuhlisiwe ukuze ukunyanga isifo Fabry. Oku kubanga nokushisa, intliziyo banzi, iingxaki zezintso. Ehlabathini lonke, abantu abanengxaki kwesi sifo, akukho abantu namawaka amathathu ngaphezu.

Le Arcalyst kweziyobisi

Abanye bakholelwa ukuba imali eninzi kakhulu ehlabathini yamayeza - umvelisi ichiza Regeneron ekuthiwa Arcalyst, kuba unyango lwe Syndrome Muckle-Wales. Pathology into ephindaphindekayo ngumkhuhlane ngakanye, amalungu abe buhlungu, zezintso. Sifo sichaphazela malunga nezigidi ezibini abantu ehlabathini lonke.

Iindleko ichiza malunga amabini anamanci mabini amawaka eerandi.

Le Soliris kweziyobisi

Abanye kwexesha eyadlulayo, iyeza kuyabiza kakhulu ehlabathini - Soliris. Ixabiso ichiza of malunga nesiqingatha sesigidi seerandi. Kuthethwa ephatha Isifo apho amajoni omzimba siyitshabalalise iiseli ezibomvu. Eli yeza kuthathwa amadoda amalunga namawaka amahlanu.

WamaYeza kunye Elaprase Naglazyme

Kwelinye ichiza ukusuka kudidi iyeza kuyabiza kakhulu ehlabathini - yinto Elaprase iziyobisi. ixabiso layo angamakhulu amane amawaka eerandi. Iindlela eboniswe Hunter syndrome. Kuba ezimbi ebalasele ukwanda ulwimi, ukonakala kwengqondo kunye nempumlo Walungisa. Ehlabathini lonke, eli chiza kuthatha malunga namawaka amabini abantu.

Malunga ixabiso efanayo Naglazyme. Ku emiselweyo ukunyanga Namiq Q-Lamy. Syndrome kuchaphazela ngaphezu kokuba abantu eziliwaka ehlabathini lonke. Kuba sifo lalidume imilebe zamafu, elifutshane ngesiqu, nentloko enkulu. Kakade ukususela kwiminyaka izigulane elinesihlanu kwisitulo sezigulana esinamavili.

Cinryze

Eli chiza - kwaye iyeza kuyabiza kakhulu kweli hlabathi into ebaluleke ngaphezu kwamakhulu amathathu amawaka eerandi. Ichiza yenzelwe ukunyanga uhlobo angioedema amafa. Kule ukugula wabonakalisa nkqubo kakuhle omzimba, ebangela Ukudumba kakhulu komlomo, kwisisu.

Iyeza kuyabiza

Ngoko ke ngubani iyeza kuyabiza kakhulu ehlabathini? Ingaba kubiza malini? iinkampani ezimbini amazwe ziye iyeza kuyabiza kakhulu ehlabathini. Glybera ibizwa ngokuba. Iindleko zonyango eli ziko liya kuba ngaphezu kwesigidi euro.

iyeza elitsha gene lenzelwe ukuba ukunyanga isifo esinqabileyo yemfuza - lipoprotein lipase intswelo. I-enzyme ilawula umgangatho lipids emzimbeni. Ngenxa gene ukuguquka enzyme nto yenziweyo umzimba, kuba has a emngciphekweni omkhulu occlusion nemithambo.

Enye igutyana ichiza liya kuxabisa ngaphezulu amahlanu amawaka euro, kwaye ikhosi epheleleyo yonyango lwesifo iya kuba ngaphezu kwesigidi. izigulane bakuthathe nale syndrome, kukho malunga namakhulu amabini ngabantu e Europe, ngoko ke isixhobo akunakwenzeka ukuba naziphina iimpembelelo kwi-mali zamaziko yempilo karhulumente.

Nangona ixabiso eliphezulu, sisixhobo nako ukumphilisa ngokupheleleyo ngezifo. Ngokutsho ababhekisi phambili, ikhondo unyango kunye zitshiphu therapy ezininzi enzyme replacement.

Ichiza Glybera gene lelona ziko yesine ehlabathini. zaye I ezimbini zokuqala eyenziwe zizazinzulu waseMerika ziveliswa China. Zombini ezi zixhobo zenzelwe ukuba unyango umhlaza, oko kukuthi, umhlaza womlomo wesibeleko kunye nangezilonda yentloko. iindlela Okwesithathu eRussia. Esi sixhobo ubizwa ngokuba "Neovaskulgen", yenzelwe yonyango lomzimba ischemia elisezantsi. Ikhosi konyango nale iyeza iya kuxabisa malunga namakhulu amathathu amawaka engange.

arhente ze-biotechnology bakholelwa ukuba amachiza nto ibiza kakhulu ehlabathini, iifoto apho zifumaneka kwinqaku obagwebelayo ngokwaso. Enyanisweni, de kufike kwala machiza, abantu abaninzi bathwaxwa ezimbi kakhulu, abakwazanga ukuphelisa izifo zabo. Iinkampani eziXuba amaYeza ke buyanceda athengise la machiza, ekubeni iintengiso zabo mfuneko ukuchitha imali. Kwakhona la machiza phantse akukho khuphiswano, nto leyo yenza abenzi ngokwabo ukumisela iindleko ichiza kunye nokufumaneka kwayo.

Kwi-inshorensi United States iinkampani kuzihlawula iindleko amachiza axabiso kwizigulane, yaye abanye abenzi sinikele khulula iyeza abasweleyo.

Iingcali kulo lonke ihlabathi zokuzigwebela iindleko eziphezulu kwamachiza amatsha. Ngokutsho kwabo, ukusetyenziswa amayeza ezinjalo ukuba izigulane ezininzi ngokuyinxenye, kwaye kwezinye iimeko ukubuyisa ngokupheleleyo ndahlukane syndromes esinganyangekiyo. Iingcali zikholelwa ukuba ixabiso la machiza iye yehla, ukhuphiswano efunekayo. bambalwa kakhulu abantu bacinga ukuba abo baye ezichongiweyo izifo kakhulu, yaye besebancinane, abakwazi ukuba babe nako ukuthenga iziyobisi, zidla amawaka. Kwiimeko ezininzi, abantu abanalo ithuba ukuze bathenge imithi eninzi ezinjalo.