Icyizere gishya kuri Duchenne Muscular Dystrophy (DMD): Reka twige ku muti witwa Duvyzat?

Ndumva ukuntu biremereye kandi biteye ubwoba iyo muganga wawe akubwira ko umwana wawe afite Duchenne muscular dystrophy (DMD). Iyi ni indwara iterwa n'uturemangingo ifata cyane cyane abahungu kandi igatuma imitsi yabo igabanuka buhoro buhoro. Ibimenyetso bikunze kugaragara mu bwana. Nta muti wuzuye wa DMD uraboneka kugeza ubu. Ariko ntugahangayike. Siyansi y'ubuvuzi iri gutera imbere umunsi ku wundi. Hamwe n'iryo terambere, icyizere gishya ni uyu muti mushya witwa Duvyzat. Reka tubiganireho mu buryo bworoshye.

Duvyzat ni iki? Ikora ite?

Mu magambo make, imitsi yacu ikenera kwitabwaho buri gihe kugira ngo ikomeze gukomera. Tekereza ku mitsi yo mu mubiri wacu nk'urukuta rw'amatafari. Kugira ngo uru rukuta rukomeze gukomera no gufatana, rukeneye urwego rwiza rwa morti. Mu buryo nk'ubwo, kugira ngo uturemangingo tw'imitsi yacu dukomeze gukomera, poroteyine yihariye yitwa dystrophine , imeze nk'morti, ni ngombwa.

Duchenne muscular dystrophy (DMD) ni indwara iterwa n'uturemangingo (genetic disorder) ibaho iyo umubiri udakora poroteyine ya dystrophin ihagije, ntuyikore ihagije, cyangwa ntuyikore ihagije. Iyo iyi 'kole' ibuze, urukuta rw'imitsi ruracika intege, rurasenyuka kandi ntirushobora gusanwa. Niyo mpamvu imitsi icika intege uko igihe kigenda gihita.

Uretse iki kibazo, abana barwaye DMD bafite imisemburo ikora cyane yitwa histone deacetylases (HDACs) . Ibi bitera ububyimbirwe buhoraho mu mitsi. Ibi bigabanya cyane gusana imitsi, kandi imitsi igasimburwa n'ibinure n'inkovu.

Kugeza ubu, abaganga bakoresheje imiti ya steroide, ubuvuzi bw'umubiri, n'ibikoresho by'ubufasha nk'intebe z'abamugaye mu kuvura indwara zo mu mutwe. Ariko Duvyzat ni wo muti wa mbere utari uwa steroide. Ukora ugenzura enzyme ya HDAC ikora cyane. Ibi bigabanya ububyimbirwe mu mitsi kandi bigatuma icika intege. Ibi bivuze ko umuvuduko w'imitsi icika intege ugabanuka.

Uyu muti nkwiye kuwukoresha nte?

Duvyzat ni umuti w'amazi unywa. Ugomba gufatwa kabiri ku munsi, mu gitondo na nijoro, hamwe n'ibiryo .

• Ni ngombwa cyane gufata umuti mu gihe kimwe buri munsi .
• Mbere yo gukoresha icupa ry'umuti, ugomba kurizunguza neza byibuze amasegonda 30 .
• Pima umuti, ukoresheje syringe yihariye iza na wo.
• Icy'ingenzi kurushaho: Ntukavange uyu muti n'amazi cyangwa ikindi kintu icyo ari cyo cyose cy'amazi.

Muganga wawe ashobora kuguha uyu muti wenyine, cyangwa akawutanga hamwe na steroid. Uzafatwa hakurikijwe uko umwana ameze.

Ni gute imiterere y'uyu muti yizwe?

Mu bushakashatsi bukomeye bwakozwe kugira ngo harebwe imikorere n'umutekano w'uyu muti, abahungu barwaye DMD bagabanyijwemo amatsinda abiri.

1. Itsinda rya mbere: Bahawe umuti witwa Duvyzat.

2. Itsinda rya 2: Bahawe ikintu gisa n'umuti, ariko nta kintu gikora kirimo. Mu buvuzi, ibi tubyita 'placebo' .

Ibi bikorwa kugira ngo harebwe ingaruka nyazo z'uyu muti, uburyo utandukanye no kuba uhari gusa. Abitabiriye ubu bushakashatsi bose bari abahungu. Impuzandengo y'imyaka yabo yari imyaka 9.

Nyuma yo gutanga ubu buvuzi mu gihe cy'ibyumweru 72, hapimwe impinduka mu bushobozi bw'abana. Hari n'ibindi bizamini byinshi byakoreshejwe kuri ibi.

• Amanota y'isuzumabumenyi ry'indwara zo mu mutwe (NSAA) ku nyota y'Amajyaruguru: Uburyo bwo gupima ubushobozi bw'umuntu urwaye indwara zo mu mutwe zo mu mutwe mu gukora imirimo ya buri munsi.
• Ikizamini cy'iminota itandatu yo kugenda n'amaguru: Kireba aho ushobora kugenda n'amaguru mu minota 6.
• Ibizamini bya MRI: Bipima impinduka mu miterere y'ibinure mu mitsi yo mu kibero.

Ni ibihe bisubizo by'ubushakashatsi?

Ibisubizo birashimishije cyane. DMD ni indwara aho ibimenyetso bigenda birushaho kwiyongera uko igihe kigenda gihita. Icyagaragaye muri ubu bushakashatsi ni uko igipimo cyo kwangirika kw'abana bafataga Duvyzat cyagabanutse cyane ugereranyije n'abana batafashe uyu muti (placebo) .

• Imbaraga n'uburyo bwo kugenda: Igihe cyakoreshejwe kugira ngo bazamuke ingazi enye cyiyongereyeho amasegonda 1.25 gusa nyuma y'ibyumweru 72 ku bana bafashe Duvyzat. Ariko byatwaye amasegonda agera kuri 3 ku bana batafashe uyu muti. Ibyo bivuze ko Duvyzat yagabanyije ubushobozi bwabo bwo kuzamuka ingazi ho 40%.
• Amanota ya NSAA: Mu mpera z'ubushakashatsi, kugabanuka kw'amanota ya NSAA kwari kugabanuka gukomeye cyane ku bana bafashe Duvyzat ugereranyije n'abatafashe uyu muti. Ibi kandi bigaragaza ko igipimo cyo kugabanuka kw'imbaraga z'imitsi cyagabanutse.

Ibi bisubizo bishobora gutandukana ku mwana wawe, bityo rero ugomba kuganira na muganga wawe ku byiza n'ingaruka z'uyu muti mbere yo gufata icyemezo.

Ingaruka mbi n'uburyo bwo kuzirwanya?

Kimwe n'indi miti iyo ari yo yose, Duvyzat ishobora kugira ingaruka mbi. Ni ngombwa kubimenya.

Ibi si byo bimenyetso byonyine. Niba ubonye ibindi bimenyetso bidasanzwe, ganira na muganga wawe ku byerekeye ibyo bimenyetso.

Ese hari ibibazo bishobora kubaho ku yindi miti?

Yego, Duvyzat ishobora kugira ingaruka ku yindi miti. Kubwibyo, ni ngombwa kubwira muganga wawe imiti yose umwana wawe afata, harimo imiti yanditswe na muganga, imiti igurishwa na muganga (OTC), vitamine, n'ibikomoka ku bimera.

By'umwihariko, menyesha muganga wawe niba ufata imiti ikurikira:

• Imiti imwe igabanya cholesterol (urugero: atorvastatin, simvastatin)
• Imiti ya diyabete nka metformin
• Imiti igira ingaruka ku mikorere y'umutima (urugero: amiodarone)
• Amwe mu matibayotike (urugero: azithromycin, ciprofloxacin)
• Imiti imwe n'imwe ivura indwara zo mu mutwe
• Imiti imwe n'imwe ivura allergie

Uru rutonde ntirwuzuye, bityo ni byiza kubwira muganga wawe ibyo umwana wawe afata byose.