Chiyembekezo chatsopano cha Duchenne Muscular Dystrophy (DMD): Tiyeni tiphunzire za mankhwala a Duvyzat?

Ndikumvetsa momwe zimakhalira zovuta komanso zoopsa dokotala wanu akakuuzani kuti mwana wanu ali ndi Duchenne muscular dystrophy (DMD). Uwu ndi matenda a majini omwe amakhudza kwambiri anyamata ndipo pang'onopang'ono amafooketsa minofu yawo. Zizindikiro nthawi zambiri zimawonekera ali ana aang'ono. Palibe mankhwala athunthu a DMD pakadali pano. Koma musadandaule. Sayansi ya zamankhwala ikupita patsogolo tsiku ndi tsiku. Ndi kupita patsogolo kumeneko, chiyembekezo chatsopano ndi mankhwala atsopano otchedwa Duvyzat. Tiyeni tikambirane mwachidule.

Kodi Duvyzat ndi chiyani? Imagwira ntchito bwanji?

Mwachidule, minofu yathu imafunika kusamalidwa nthawi zonse kuti ikhale yolimba. Taganizirani za minofu m'thupi mwathu ngati khoma la njerwa. Kuti khoma ili likhale lolimba komanso logwirizana, limafunika matope abwino. Mofananamo, kuti maselo athu a minofu akhale olimba, puloteni yapadera yotchedwa dystrophin , yomwe ili ngati matope, ndi yofunika.

Duchenne muscular dystrophy (DMD) ndi matenda omwe amapezeka m'thupi pamene thupi silipanga mapuloteni okwanira a dystrophin, silipanga okwanira, kapena silipanga okwanira. Pamene 'guluu' uyu watayika, khoma la minofu limakhala lofooka, limasweka ndipo silingathe kukonzedwa. Ndicho chifukwa chake minofu imafooka pakapita nthawi.

Kuwonjezera pa vutoli, ana omwe ali ndi DMD ali ndi ma enzyme ochulukirapo otchedwa histone deacetylases (HDACs) . Izi zimayambitsa kutupa kosatha m'minofu. Izi zimachepetsanso kukonzanso minofu, ndipo minofu imalowedwa m'malo ndi mafuta ndi minofu ya zipsera.

Mpaka pano, madokotala akhala akugwiritsa ntchito ma steroid, physiotherapy, ndi zipangizo zothandizira monga mipando ya olumala pochiza matenda amisala. Koma Duvyzat ndi mankhwala oyamba osagwiritsa ntchito ma steroid. Amagwira ntchito polamulira enzyme ya HDAC yomwe imagwira ntchito mopitirira muyeso. Izi zimachepetsa kutupa m'minofu ndikulola kuti iwonongeke. Izi zikutanthauza kuti liwiro lomwe minofu imafooka limachepetsedwa.

Kodi ndiyenera kugwiritsa ntchito bwanji mankhwalawa?

Duvyzat ndi mankhwala amadzimadzi omwe mumamwa. Ayenera kumwedwa kawiri patsiku, m'mawa ndi usiku, pamodzi ndi chakudya .

• Ndikofunikira kwambiri kumwa mankhwala nthawi yomweyo tsiku lililonse .
• Musanagwiritse ntchito botolo la mankhwala, muyenera kuligwedeza bwino kwa masekondi osachepera 30 .
• Yesani mankhwalawo pogwiritsa ntchito sirinji yapadera yomwe imabwera nayo.
• Chofunika kwambiri: Musasakanize mankhwalawa ndi madzi kapena madzi ena aliwonse.

Dokotala wanu angapereke mankhwalawa yekha, kapena angapereke limodzi ndi ma steroids. Zidzasankhidwa kutengera momwe mwanayo alili.

Kodi makhalidwe a mankhwalawa aphunziridwa bwanji?

Mu kafukufuku wamkulu wofufuza momwe mankhwalawa amathandizira komanso chitetezo chake, anyamata omwe ali ndi DMD adagawidwa m'magulu awiri.

1. Gulu loyamba: Anapatsidwa mankhwala otchedwa Duvyzat.

2. Gulu lachiwiri: Anapatsidwa chinthu chomwe chimawoneka ngati mankhwala, koma chinalibe chogwiritsira ntchito. Mu zamankhwala, timatcha ichi kuti 'placebo' .

Izi zimachitika kuti aone ngati mankhwalawa ali ndi mphamvu yeniyeni, kusiyana kwake ndi kungokhalapo. Onse omwe adachita nawo kafukufukuyu anali anyamata. Avereji ya zaka zawo inali zaka 9.

Pambuyo popereka chithandizochi kwa milungu 72, kusintha kwa luso la ana kunayesedwa. Mayeso ena angapo anagwiritsidwa ntchito pa izi.

• Chigoli cha North Star Ambulatory Assessment (NSAA): Njira yoyezera luso la munthu amene ali ndi matenda a shuga (DMD) pochita zinthu za tsiku ndi tsiku.
• Mayeso oyenda kwa mphindi zisanu ndi chimodzi: Amaona mtunda womwe mungayendemo mu mphindi zisanu ndi chimodzi.
• Mayeso a MRI: Amayesa kusintha kwa mafuta omwe ali m'minofu ya ntchafu.

Kodi zotsatira za kafukufukuyu zinali zotani?

Zotsatira zake n’zolimbikitsa kwambiri. Matenda a shuga ndi matenda omwe zizindikiro zake zimakula pakapita nthawi. Chomwe chinali chodziwikiratu mu kafukufukuyu chinali chakuti kuchuluka kwa ana omwe adamwa Duvyzat kunali kocheperako poyerekeza ndi ana omwe sanamwe mankhwalawo (placebo) .

• Mphamvu ndi kuyenda: Nthawi yomwe inatenga kukwera masitepe anayi inawonjezeka ndi masekondi 1.25 okha patatha milungu 72 mwa ana omwe adatenga Duvyzat. Koma zinatenga masekondi atatu kupitirira apo mwa ana omwe sanatenge mankhwalawa. Izi zikutanthauza kuti Duvyzat anachepetsa kuchepa kwa mphamvu zawo zokwera masitepe ndi pafupifupi 40%.
• Zigoli za NSAA: Kumapeto kwa kafukufukuyu, kuchepa kwa zigoli za NSAA kunali kochepa kwambiri mwa ana omwe adatenga Duvyzat poyerekeza ndi omwe sanatenge mankhwalawa. Izi zikusonyezanso kuti kuchuluka kwa mphamvu ya minofu kunachepa.

Zotsatirazi zingasiyane kwa mwana wanu, choncho muyenera kukambirana za ubwino ndi zoopsa za mankhwalawa ndi dokotala wanu musanapange chisankho.

Zotsatirapo zake ndi momwe mungathanirane nazo?

Monga mankhwala ena aliwonse, Duvyzat ikhoza kukhala ndi zotsatirapo zake. Ndikofunikira kudziwa izi.

Izi si zizindikiro zokhazo. Ngati mukukumana ndi zizindikiro zina zachilendo, lankhulani ndi dokotala wanu za izo.

Kodi pangakhale mavuto ndi mankhwala ena?

Inde, Duvyzat imatha kuyanjana ndi mankhwala ena. Chifukwa chake, ndikofunikira kuuza dokotala wanu za mankhwala onse omwe mwana wanu akumwa, kuphatikizapo mankhwala olembedwa ndi dokotala, mankhwala ogulitsidwa kunja kwa chipatala (OTC), mavitamini, ndi mankhwala azitsamba.

Makamaka, dziwitsani dokotala wanu ngati mukumwa mankhwala otsatirawa:

• Mankhwala ena ochepetsa mafuta m'thupi (monga atorvastatin, simvastatin)
• Mankhwala a shuga monga metformin
• Mankhwala omwe amakhudza kayendedwe ka mtima (monga amiodarone)
• Maantibayotiki ena (monga azithromycin, ciprofloxacin)
• Mankhwala ena a matenda amisala
• Mankhwala ena a ziwengo

Mndandandawu sunathe, choncho ndi bwino kuuza dokotala wanu zonse zomwe mwana wanu akumwa.