Ko iwe unodana kupfuura zvinodhura mushonga munyika?

Zveminda mamwe makambani anogadzira harikunguruswi pamusoro bhiriyoni. Izvi imhaka yokuti vachikukumikidzai zvinodhaka zvinodhura, kugadzirwa batch diki. Kazhinji mishonga iyi, kudzivisa isingawanzowanikwi pathologies. Dzimwe nyanzvi dzinofunga kuti mari vamwe mishonga akanyanya kukwirira nokuti hakuna makwikwi. A pamazita zvigadzirwa zvakadaro rakanyatsojeka yakakura, pakati pavo zvikuru zvinodhura munyika mushonga kuchaita mari kupfuura miriyoni euros.

The mushonga Aldurazyme

mushonga uyu chakagadzirirwa kurapa Hurler urwere, izvo kunooneka pacharo muuduku. Iye achitambura paurwere vanhu zvishoma pane mazana matanhatu pasi pose. Chirwere kuchititadzisa mumuviri uye tsika kukura kwevana vaduku sezvo mana nemakore.

Mari Zvivako izvi anenge zviuru mazana maviri emadhora.

Drug "Cerezyme"

Ngozi zvinodhaka ichi mari pamusoro zvakafanana sezvo Aldurazyme. "Cerezyme" - zvakangofanana kupfuura zvinodhura mushonga munyika. Zvinonzi nachiremba Gaucher chirwere. Nokuti chirwere dzinoratidzwa kuunganidza mafuta deposits mumapapu, chiropa, pfungwa uye mwongo. mhando iyi rinooma kufutisa yakaoma kushanda mumapapu neitsvo. Mishonga unotsiva enzayemu iri Gaucher chirwere chose vasipo.

Munyika yose, vanhu vane ichi chirwere vanenge zviuru zvitanhatu vanhu.

The mushonga Myozyme

Pane kuda mazana zvinodhaka, mutengo vanopfuura chiuru euros por chinu. zvinodhaka zvose izvi ndezvaJehovha muchikwata che "vakawanda mushonga zvinodhura munyika." Ko zita rimwe mushonga muboka iri? mishonga iyi Myozyme, nokuti mishonga Pompe syndrome. A zvokurwara dzinoratidzwa kushayiwa Alpha-glucosidase. Izvi zvinotungamirira kuparadzwa tsandanyama rakapinza kuwedzera bhuku mwoyo, kuva kufema zvokurwara. Kakawanda, kurwara iri dzakaonekwa muuduku.

Kushandiswa zvinodhaka chingamisa kwemasayendisiti uye unobatsira vana vanotanga kufema vakazvimiririra. Mari kurapwa mumiririri achava arasikirwe zviuru zana euros kuti mwana. Kana maperembudzi chirwere vakura, ipapo kurapwa achatora anopfuura mazana zviuru zvitatu.

zvinoreva Fabrazyme

Mumwe mushonga kupfuura zvinodhura munyika - payo inzira Fabrazyme. Mari kurapika kuchaita mari inenge zvitatu namazana zvemadhora.

The chinodhaka akagadzirirwa mishonga Fabry chirwere. Chinokonzera kutekenyedzwa kutsva, ane wakakurisa mwoyo, itsvo matambudziko. Munyika yose, vanhu vanotambura nezvirwere ichi, hapana kupfuura vanhu zviuru zvitatu.

The mushonga Arcalyst

Vamwe vanodavira kuti zvikuru zvinodhura munyika zvokurapa - zvinodhaka mugadziri Regeneron akadana Arcalyst, nokuti pakurapa Syndrome Muckle-Wales. Zvokurwara ari nekorasi inoratidza fivha, kurwadza kwemajoini, itsvo. Chirwere inobata vanenge mamiriyoni maviri vanhu pasi pose.

Mari zvinodhaka anenge maviri namakumi maviri nezviviri zvemadhora.

The mushonga Soliris

Vamwe nguva kare, kupfuura zvinodhura mushonga munyika - Soliris. Mutengo mushonga vanenge hafu yemiriyoni emadhora. Zvinoreva kubata chirwere umo zvirwere zvinoparadza masero matsvuku eropa. mushonga uyu akatorwa varume vanenge zvuru zvishanu.

Mishonga Elaprase Naglazyme

Chimwe mushonga kubva muchikwata zvikuru zvinodhura mushonga munyika - iri chinodhaka Elaprase. mutengo wayo zviuru zvina mazana zvamadhora. Means akaratidza kuti Hunter syndrome. For pathologies dzinoratidzwa kuwedzera mutauro, uropi uye ndokudzora mhino. Around nyika, chinodhaka ichi zvinotora zvuru zviviri vanhu.

Inenge chete mutengo Naglazyme. Zvinonzi ushandiswe kubata Maroto-Lamy. Syndrome inokanganisa vanodarika chiuru vanhu pasi pose. Nokuti chirwere dzinoratidzwa miromo paukobvu, nomumhu uye musoro guru. Nechekare vanamakore gumi varwere muwiricheya.

Cinryze

chinodhaka ichi - kupfuura zvinodhura mushonga munyika uye unokosha kupfuura zviuru mazana matatu zvemadhora. The mushonga chakagadzirirwa kubata nhaka angioedema mhando. In chirwere ichi akaratidza havachanyatsogoni rokuzvidzivirira, hunokonzera yakanyanya mumatenhere miromo, asi abdominal mhango.

The mushonga kupfuura zvinodhura

Saka chii mushonga kupfuura zvinodhura munyika? anoita kunzanga zvakawanda sei? Makambani maviri vatorwa zvave mushonga kupfuura zvinodhura munyika. Glybera inonzi. Mari kurapwa chivako ichi ichava euros vanopfuura miriyoni.

New Gene mushonga rinotarisirwa kubata isingawanzowanikwi nemagene chirwere - lipoprotein lipase kukanganisika. The enzayemu anoita pachiyero lipids pamuviri. Nokuda Gene mutation enzayemu haisi zvinoberekwa muviri, nekuti ine mukuru ngozi vascular occlusion.

Imwe chinu mushonga achava mari vanopfuura zviuru makumi mashanu euro, uye azere yawapiwa zvirwere richava zvinopfuura miriyoni. Varwere kuzova pamwe chirwere ichi, pane vanhu vanenge mazana maviri muEurope, saka chokushandisa angangotadza risingakoshi nezvainoita bhajeti evoruzhinji utano masangano.

Pasinei mubhadharo, kuti chokushandisa anokwanisa kuporesa zvokurwara zvachose. Maererano Developers, yawapiwa pamwe isingadhuri mazhinji enzayemu yokutsiva mishonga.

The mushonga Glybera jini nzvimbo chechina munyika. maviri okutanga yakagadzirwa nemasayendisiti American uye dzikabereka mu China. Both zvokushandisa zvakagadzirirwa pakurapa kenza, kureva, gomarara uye mapundu musoro. kunoreva wechitatu Russia. chokushandisa ichi anonzi "Neovaskulgen", aiendeswa pakurapa akaderera richiripo ischemia. Yawapiwa pamwe mishonga ino mari inenge zvitatu namazana rubles.

Masangano noutachiona vanodavira kuti mishonga kupfuura zvinodhura munyika, mapikicha rinogona kuwanikwa munyaya unoruramisa pacharo. Kutaura zvazviri, kusvikira veMubatanidzwa zvinodhaka izvi, vanhu vazhinji nechirwere chakakomba pathologies, vakanga vasingakwanisi kubvisa zvirwere zvavo. Makambani Pharmaceutical hazvibatsiri kutengesa zvinodhaka izvi, sezvo shambadzo dzavo havafaniri kushandisa mari. Uyewo zvinodhaka izvi ndizvo zvinenge hapana makwikwi, iyo inoita vagadziri pachavo kuona mari zvinodhaka uye ayo Kuwanikwa.

MuUnited States inishuwarenzi makambani dzosera mari mishonga anodhura varwere, uye dzimwe vagadziri kupa pachena mishonga vanoshayiwa.

Nyanzvi munyika yose kupembedza kwemitengo mishonga mitsva. Maererano navo, kushandisa mishonga yakadaro kubvumira varwere zhinji zvishomanana, uye mune zvimwe zviitiko kupora zvachose ndichipfeka kubvisa chisingarapiki syndromes. Nyanzvi dzinodavira kuti mutengo mishonga iyi yanga ichidzikira, makwikwi kunodiwa. Chete vashoma vanhu vanofunga kuti avo vanozivikanwa chirwere chakakomba, uye kunyange vachiri vaduku, kashoma kuti kukwanisa kutenga zvinodhaka, kukudyira zviuru. Kazhinji, vanhu havana mukana mishonga dzinodhura kwakadaro.