Ngiyaqonda ukuthi kunzima futhi kwesabeka kangakanani lapho udokotela wakho ekutshela ukuthi ingane yakho ine-Duchenne muscular dystrophy (DMD). Lesi yisifo sofuzo esithinta kakhulu abafana futhi kancane kancane senza imisipha yabo ibe buthaka. Izimpawu zivame ukubonakala ebuntwaneni. Akukho ukwelashwa okuphelele kwe-DMD okwamanje. Kodwa ungakhathazeki. Isayensi yezokwelapha ithuthuka usuku nosuku. Ngaleyo ntuthuko, ithemba elisha yilo muthi omusha obizwa ngokuthi i-Duvyzat. Ake sixoxe ngakho kalula.
Iyini i-Duvyzat? Isebenza kanjani?
Kalula nje, imisipha yethu idinga ukunakekelwa njalo ukuze ihlale iqinile. Cabanga ngemisipha emzimbeni wethu njengefana nodonga lwezitini. Ukuze lolu donga luhlale luqinile futhi luhlangene, ludinga ungqimba oluhle lwe-mortar. Ngokufanayo, ukuze amangqamuzana emisipha yethu agcine eqinile, iphrotheni ekhethekile ebizwa ngokuthi i-dystrophin , efana ne-mortar, ibalulekile.
I-Duchenne muscular dystrophy (DMD) yisimo sofuzo esenzeka lapho umzimba ungakhiqizi iphrotheni ye-dystrophin eyanele, ungakhiqizi eyanele, noma ungakhiqizi eyanele. Uma le 'glue' ilahlekile, udonga lwemisipha luba buthakathaka, luqhekeke futhi alukwazi ukulungiswa. Yingakho imisipha iba buthakathaka ngokuhamba kwesikhathi.
Ngaphezu kwale nkinga, izingane ezine-DMD zinama-enzyme asebenza ngokweqile abizwa ngokuthi ama-histone deacetylases (ama-HDAC) . Lokhu kubangela ukuvuvukala okungapheli emisipheni. Lokhu kunciphisa kakhulu ukulungiswa kwemisipha, futhi imisipha ithathelwa indawo ngamafutha nezicubu ezibomvu.
Kuze kube manje, odokotela bebesebenzisa ama-steroid, ukwelashwa ngokomzimba, kanye namadivayisi okusiza njengezihlalo zabakhubazekile ukwelapha i-DMD. Kodwa i-Duvyzat umuthi wokuqala ongeyona i-steroidal. Isebenza ngokulawula i-enzyme ye-HDAC esebenza ngokweqile. Lokhu kunciphisa ukuvuvukala emisipheni futhi kuyivumele ukuthi iphuke. Lokhu kusho ukuthi ijubane lapho imisipha iba buthaka khona liyehliswa.
Ngingawusebenzisa kanjani lo muthi?
I-Duvyzat umuthi owuketshezi owuphuza ngomlomo. Kufanele uthathwe kabili ngosuku, ekuseni nasebusuku, kanye nokudla .
- Kubaluleke kakhulu ukuphuza umuthi ngesikhathi esifanayo nsuku zonke .
- Ngaphambi kokusebenzisa ibhodlela lomuthi, kufanele ulishukumise kahle okungenani imizuzwana engama-30 .
- Kala umuthi, usebenzisa isirinji esikhethekile esihambisana nawo.
- Okubaluleke kakhulu: Ungawuxubi lo muthi namanzi noma olunye uketshezi.
Udokotela wakho angakunikeza lo muthi eyedwa, noma angakunikeza kanye nama-steroid. Kuzonqunywa kuye ngesimo sengane.
Izakhiwo zalo muthi ziye zafundwa kanjani?
Esifundweni esikhulu sokuhlola ukusebenza kahle nokuphepha kwalesi sidakamizwa, abafana abane-DMD bahlukaniswe ngamaqembu amabili.
1. Iqembu lokuqala: Banikwa umuthi iDuvyzat.
2. Iqembu 2: Banikwa into efana nomuthi, kodwa engenaso isithako esisebenzayo kuwo. Kwezokwelapha, lokhu sikubiza ngokuthi 'i-placebo' .
Lokhu kwenzelwa ukukala umphumela wangempela womuthi, ukuthi uhluke kangakanani ekubeni lapho nje. Bonke abahlanganyeli kulolu cwaningo babengabafana. Isilinganiso sabo seminyaka sasiyiminyaka eyi-9 ubudala.
| Izindlela eziyinhloko zokufaneleka zokufakwa ocwaningweni | |
|---|---|
| Ukuhlolwa | Isidingo |
| Ukukhuphuka ngezitebhisi ezine | Ingane inikwe isikhathi sokukhuphuka izinyathelo ezine ngokushesha okukhulu. Lokhu kufanele kuthathe ngaphansi kwemizuzwana eyisishiyagalombili. |
| Isikhathi sokuvuka | Isikhathi esithathwayo ukusukuma ulele phansi ngaphandle kosizo siyalinganiswa. Lokhu kufanele kuthathe phakathi kwemizuzwana emi-3 neyi-10. |
Ngemva kokunikeza lokhu kwelashwa amasonto angu-72, kwalinganiswa izinguquko emakhonweni ezingane. Kwasetshenziswa ezinye izivivinyo eziningana kulokhu.
- Isilinganiso se-North Star Ambulatory Assessment (NSAA): Indlela yokulinganisa ikhono lomuntu one-DMD lokwenza imisebenzi yansuku zonke.
- Ukuhlolwa kokuhamba ngezinyawo kwemizuzu eyisithupha: Kubona ukuthi ungahamba ibanga elingakanani ngemizuzu eyisithupha.
- Ukuhlolwa kwe-MRI: Kukala izinguquko ekufakweni kwamafutha emisipheni yendawo yethanga.
Yaba yini imiphumela yocwaningo?
Imiphumela iyakhuthaza ngempela. I-DMD yisimo lapho izimpawu ziba zimbi khona ngokuhamba kwesikhathi. Okwakucacile kulolu cwaningo ukuthi izinga lokuwohloka kwezingane ezithathe i-Duvyzat lalihamba kancane kakhulu kunezingane ezingazange zithathe umuthi (i-placebo) .
Lokhu kusho ukuthi nakuba lo muthi ungeke welaphe ngokuphelele lesi sifo, unganciphisa izinga lobuthakathaka bemisipha futhi usize ingane ukuthi ilondoloze ikhono layo lokusebenza isikhathi eside.
- Amandla nokuhambahamba: Isikhathi esathathwa ukukhuphuka izitebhisi ezine sanda ngemizuzwana eyi-1.25 kuphela ngemva kwamasonto angama-72 kubantwana abathathe iDuvyzat. Kodwa kwathatha cishe imizuzwana emi-3 ngaphezulu kubantwana abangazange bathathe umuthi. Lokho kusho ukuthi iDuvyzat yehlisa ukwehla kwamandla abo okukhuphuka izitebhisi cishe ngo-40%.
- Amaphuzu e-NSAA: Ekupheleni kocwaningo, ukwehla kwamaphuzu e-NSAA kwakuncane kakhulu ezinganeni ezithathe i-Duvyzat kunalezo ezingazange zithathe umuthi. Lokhu futhi kubonisa ukuthi izinga lokwehla kwamandla emisipha lehlile.
Le miphumela ingahluka enganeni yakho, ngakho-ke kufanele uxoxe ngezinzuzo nezingozi zalo muthi nodokotela wakho ngaphambi kokwenza isinqumo.
Imiphumela emibi nokuthi ungayiphatha kanjani?
Njenganoma yimuphi umuthi, i-Duvyzat ingaba nemiphumela emibi. Kubalulekile ukuqaphela lokhu.
| Umphumela ongemuhle | Okudingeka ukwenze |
|---|---|
| Imiphumela emibi evamile: - Uhudo - Ukucanuzela/ukuhlanza - Isisu esibuhlungu - Imfiva | Uma uhlangabezana nanoma yiziphi zalezi zimpawu , tshela udokotela wakho ngokushesha . Phuza amanzi amaningi kanye noketshezi. Udokotela wakho angase akuncomele imithi noma ukushintsha ukudla uma kudingeka. |
| Imiphumela emibi ephawulekayo: - Ukwehla kwama-platelet egazi (i-thrombocytopenia) - Amazinga aphezulu amafutha egazini (i-hypertriglyceridemia) | Udokotela uvame ukwenza ukuhlolwa kwegazi ukuze aqaphe lokhu. Uma uphuka kalula, uma ikhala lakho liphuma igazi, uma unenkinga yokumisa ukuphuma kwegazi,Tshela udokotela ngokushesha. |
Lezi akuzona zodwa izimpawu. Uma uhlangabezana nanoma yiziphi ezinye izimpawu ezingavamile, khuluma nodokotela wakho ngazo.
Ingabe kungaba nezinkinga ngeminye imithi?
Yebo, i-Duvyzat ingasebenzisana neminye imithi. Ngakho-ke, kubalulekile ukutshela udokotela wakho ngayo yonke imithi ethathwa yingane yakho, okuhlanganisa imithi kadokotela, imithi ethengiswa ngaphandle kwemithi kadokotela (OTC), amavithamini, kanye nemikhiqizo yamakhambi.
Ikakhulukazi, yazisa udokotela wakho uma uthatha imithi elandelayo:
- Eminye imithi yokwehlisa i-cholesterol (isib. i-atorvastatin, i-simvastatin)
- Imithi yesifo sikashukela efana ne-metformin
- Imithi ethinta isigqi senhliziyo (isb. i-amiodarone)
- Amanye ama-antibiotic (isib. azithromycin, ciprofloxacin)
- Eminye imithi yokwelapha isifo sengqondo
- Eminye imithi yokwelapha ukungezwani komzimba
Lolu hlu aluphelele, ngakho-ke kungcono ukutshela udokotela wakho ngakho konke okuthathwa yingane yakho.
Umlayezo Wokuya Nawe Ekhaya
- I-Duvyzat umuthi omusha, ongeyona i-steroidal onciphisa izinga lobuthakathaka bemisipha obubangelwa yi-Duchenne Muscular Dystrophy (DMD).
- Lokhu ngeke kuselaphe ngokuphelele lesi sifo, kodwa kuzosiza kakhulu ekuthuthukiseni izinga lempilo yengane.
- Landela imiyalelo kadokotela wakho ngqo lapho usebenzisa umuthi. Uma uhlangabezana nanoma yimiphi imiphumela emibi, yazisa udokotela wakho ngokushesha.
- Ungakhohlwa ukutshela udokotela ngayo yonke eminye imithi ingane yakho eyithathayo.
- Awuwedwa kulolu hambo. Udokotela wakho, uchwepheshe wakho wezokwelapha ngokomzimba, nomndeni wakho bonke balapha ukuzokusiza. Ungesabi.
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