Chiyembekezo chatsopano kwa iwo omwe ali ndi matenda a Fabry: Tiyeni tikambirane za mankhwala a Galafold!

Ngati inu kapena munthu amene mumamudziwa ali ndi matenda a Fabry, mwina munamvapo za mankhwala otchedwa Galafold. Ndi mankhwala oyamba komanso okhawo omwe amamwa a matenda a Fabry. Mankhwala ena a matenda a Fabry, monga mankhwala obwezeretsa ma enzyme , amaperekedwa ndi jakisoni. Galafold ndi mtundu wa mankhwala a "chaperone" omwe amagwira ntchito mosiyana kwambiri. Sigwira ntchito pa mitundu yonse ya matenda a Fabry. Komabe, ingathandize kwambiri anthu omwe ali ndi kusintha kwina mu jini komwe kumayambitsa matendawa.

Kodi matenda a Fabry ndi chiyani? Kodi Galafold amagwira ntchito bwanji?

Mwachidule, matenda a Fabry ndi matenda osowa kwambiri omwe amafalikira kuchokera kwa kholo kupita kwa mwana. Anthu omwe ali ndi matenda a Fabry ali ndi enzyme yotchedwa alpha-galactosidase A (alpha-gal) m'thupi lawo. Koma enzyme iyi sigwira ntchito bwino. Izi zili choncho chifukwa pali kusintha kwa majini komwe kumaiuza kuti ipange enzyme iyi.

Alpha-gal ndi enzyme yofunika kwambiri yomwe imapezeka m'maselo ambiri m'thupi lathu. Ntchito yake yayikulu ndikuphwanya mafuta otchedwa GL-3 . Chifukwa chake, enzyme iyi ikalephera kugwira ntchito bwino, GL-3 imayamba kusonkhana mkati mwa maselo a thupi. Pakapita nthawi, kusonkhanitsa kumeneku kungayambitse kuwonongeka kwakukulu ku ziwalo zofunika m'thupi, monga impso , mtima , khungu, ubongo, ndi dongosolo lamanjenje .

Pali mitundu iwiri ikuluikulu ya matenda a Fabry.

Pamene anthu omwe ali ndi matenda a Fabry akukalamba, GL-3 imapitirizabe kusonkhana m'maselo awo. Izi zingayambitse mavuto aakulu azaumoyo pakapita nthawi. Anthu ambiri amakhala ndi mavuto a impso, zomwe pamapeto pake zingayambitse kulephera kwa impso . Matendawa amathanso kuwononga mtima ndi mitsempha yamagazi , zomwe zimawonjezera chiopsezo cha matenda a mtima kapena sitiroko.

Anthu ambiri amapatsidwa Enzyme Replacement Therapy (ERT) pa izi. Izi zimaphatikizapo kubaya jekeseni wa enzyme yomwe imagwira ntchito bwino yomwe imapangidwa kunja kwa thupi. Thupi limatha kugwiritsa ntchito enzyme imeneyo kuphwanya GL-3.

Komabe, Galafold imagwira ntchito mosiyana kwambiri. Chogwiritsidwa ntchito mu Galafold ndi mankhwala otchedwa `migalastat`. Chomwe chimagwira ntchito ndichakuti chimalumikizana ndi enzyme ya `alpha-gal` yomwe ilipo m'thupi la wodwalayo, koma sichikhazikika. Tangoganizani kuti enzyme ya `alpha-gal` m'thupi lanu ndi 'yotupa' pang'ono, 'yosakhazikika'. Chifukwa chake, singagwire ntchito bwino. Chomwe mankhwala otchedwa Galafold amachita, monga bwenzi, amalumikizana ndi enzyme iyi 'yotupa' ndikuyikhazikitsa. Kenako enzyme imeneyo imatha kugwira ntchito yake bwino. Ichi ndi chomwe timachitcha "Chaperone Therapy" .

Kodi aliyense angagwiritse ntchito Galafold?

Ayi. Galafold ingagwiritsidwe ntchito kokha mwa anthu omwe ali ndi mtundu winawake wa kusintha komwe kungatheke mu jini yomwe imapanga enzyme ya `alpha-gal`. Enzyme ya `alpha-gal` yomwe imapangidwa ndi kusinthaku imatha kugwira ntchito, koma ndi yosakhazikika kwambiri kuti igwire ntchito bwino. Galafold imamatira ku enzyme imeneyo, kuikhazikitsa, ndikuipangitsa kuti igwire ntchito.

Odwala ena a Fabry sapanga enzyme ya `alpha-gal` konse, kapena enzyme yomwe imapangidwa sigwira ntchito konse. Galafold sidzathandiza anthu otere. Chifukwa palibe enzyme m'thupi kuti ikhazikike. Enzyme replacement therapy (ERT) ndiyo yoyenera kwa iwo.

Choncho, dokotala wanu asanakupatseni Galafold mankhwala, adzakuyesani mayeso apadera a majini kuti adziwe mtundu weniweni wa kusintha kwa majini omwe muli nawo. Izi zidzatsimikizira ngati mankhwalawa angakhale othandiza kwa inu kapena ayi.

Kodi mumagwiritsa ntchito bwanji mankhwalawa?

Gawo ili ndi lofunika kwambiri chifukwa kuti mupeze phindu lonse la mankhwala, muyenera kuligwiritsa ntchito moyenera.

• Momwe mungamwere: Galafold ndi kapisozi. Amamwedwa tsiku lililonse , kutanthauza tsiku lililonse.
• Nthawi ya tsiku: Ndikofunikira kwambiri kumwa nthawi yomweyo tsiku lililonse. Mwachitsanzo, ngati munamwa nthawi ya 8 koloko m'mawa tsiku loyamba, muyeneranso kumwa nthawi ya 8 koloko m'mawa tsiku lotsatira.
• Chofunika kwambiri: Mankhwalawa ayenera kumwedwa pamimba yopanda kanthu . Izi zikutanthauza kuti simuyenera kudya chilichonse kwa maola awiri musanayambe kumwa mankhwalawo kapena maola awiri mutamwa.
• Chinthu china chofunika: Komanso, musamwe chilichonse chokhala ndi caffeine (monga tiyi, khofi, zakumwa zina zoziziritsa kukhosi) kwa maola awiri musanayambe kumwa mapiritsi komanso maola awiri mutamwa mapiritsi. Panthawiyi, mutha kumwa madzi, zakumwa zopanda ulusi, kapena zakumwa zopanda caffeine.

Pofuna kukuthandizani kupewa kusokoneza masiku anu, mankhwalawa amabwera mu phukusi lapadera. Amatchedwa 'khadi la chithuza.' Ali ndi njira yolembera masiku. Mumachotsa khadi la chithuza tsiku lomwe mwamwa mankhwala, ndipo tsiku lomwe simunamwe mankhwalawo, mumachotsa bwalo lopanda kanthu m'malo mwake. Mwanjira imeneyi, mutha kulemba khadi tsiku lililonse osaphonya tsiku lomwe mukufuna kumwa mankhwalawo.

Kodi pali zotsatirapo zake ndi mavuto omwe amabwera chifukwa cha mankhwala ena?

Anthu ena omwe amatenga Galafold akhoza kukhala ndi chiopsezo chowonjezeka cha matenda opatsirana m'mapapo (chimfine, pakhosi) komanso matenda opatsirana m'mitsempha ya mkodzo. Njira yabwino yopewera matenda ndi kusamba m'manja pafupipafupi, kupewa anthu odwala, komanso kumwa madzi ambiri.

Kuphatikiza apo, monga tidakambirana kale, caffeineChifukwa chake, Galafold ikhoza kukhala yopanda ntchito kwenikweni. Choncho samalani ndi zinthu zomwe zili ndi caffeine. Ndikofunikanso kudziwitsa dokotala wanu za mankhwala ena aliwonse, mavitamini, kapena mankhwala achikhalidwe omwe mukumwa.