Uyini amafutha kaLornzo?

Funda nge-ALD Treatment Ephefumulelwe nguLornzo Odone

Amafutha ka-Lorenzo yindlela yokwelashwa eyenzelwe ubuntwana obuncane obunama- cerebral adrenoleukodystrophy (ALD) , isifo sengqondo se-myelin esingajwayelekile futhi esibulalayo. Kule minyaka, ukwelashwa sekutholile ukubonga okukhulu ngokubheka ucwaningo oluqhubekayo lwesayensi, i-Myelin Project, kanye nefilimu ye-1992, ama -Oil Lorenzo.

Isingeniso se-Adrenoleukodystrophy (ALD)

I-Adrenoleukodystrophy (ALD) isifo sofuzo sibangela ukwakha amafutha amaningi ase-chain chain abhubhisa i-myelin, isembozo sokuzivikela phezu kwe-neurons ebuchosheni.

Ngaphandle kwalokhu okuvikelayo, izimpawu eziyekezelayo ezifana nobuphofu, ukuzithulula, ukugunjwa, ukulahleka kwesisindo, nokucindezeleka okuqhubekayo kungenzeka.

Izimpawu ze-ALD ngokuvamile zivela phakathi kweminyaka engamashumi amane kuya kweshumi. Lesi sifo siphuthuma ngokushesha futhi sivame ukubulala eminyakeni emibili kuya kwemihlanu yesifo seqala. Ngenxa yokuthi i-ALD izuzwe njengefa, nge-X chromosome, isimo esibi kunazo zonke sesimo sithinta abafana kuphela. I-adrenal gland iyathinteka, eyaziwa ngokuthi isifo sika-Addison, ngamaphesenti angu-90 wamacala.

Uyini amafutha kaLornzo?

Ngokombiko we-Myelin Project, ama-Lorenzo ama-Oil iyinhlanganisela yenhlanganisela e-4 kuya kwe-1 ye-erucic acid ne-oleic acid, ekhishwe ngamafutha e-rapeseed namafutha omnqumo, ngokulandelana. Ukwelashwa kwamafutha, uma kuqala ekuqaleni kwabafana abangenakuqhathaniswa ne-ALD, noma abafana abangakabonakali izimpawu, kuye kwafakazelwa ukunciphisa inani lamafutha amaningi asezintweni uma kusetshenzwa nsuku zonke, okunganciphisa ukuqala kwesifo.

Kodwa-ke, ucwaningo alukwazanga ukukhombisa ukuthi amafutha ayeka ukuqhuma kwesifo futhi uyaziwa ukuthi ayiguquki noma ayilungise i-myelin eyonakalisiwe. Ukwelashwa okungcono kakhulu okwamanje okutholakala ku-ALD kungukuthi ukufakelwa komnkantsha wamathambo ngenkathi kusenjalo. Kodwa-ke, ukuguqulwa kwamathambo emasaka kuyingozi kakhulu kwiziguli ezincane ngenxa yobungozi bokutheleleka nokulahlwa.

Umfana Ophefumula Ukwelashwa

Ukwelashwa kubizwa ngokuthi "i-olin Lorenzo" ngemuva kukaLornzo Odone, umfana onama-ALD obazali bakhe, uMikhaela no-Augusto, benqaba ukwamukela ukuthi kwakungekho ukwelashwa kwe-ALD nokuthi uLorenzo uzofa ngemuva nje kokutholakala kwakhe ngo-1984. Ngokucwaninga okujulile nokuphikelela , Abazali bakaLornzo basiza ukuthuthukisa amafutha.

Ngemva kokuthatha amafutha nsuku zonke kusukela ekuthuthukiseni kwawo, u-Lorenzo wagcina esedlule ukubikezela odokotela iminyaka engaphezu kwengu-20. Ushone ngosuku olulandelayo ngemuva kokuzalwa kwakhe okungu-30 kusukela ezinkingeni ze-pneumonia. Indaba yomndeni yayidume kakhulu embukisweni we-1992, ama -Oil Lorenzo , ephethe uSusan Sarandon noNick Nolte.

Ukutholakala kwamafutha ka-Lorenzo

Amafutha ka-Lorenzo okwamanje atholakale kuphela ezigulini zase-United States ezibhalisiwe esilingo somtholampilo njengoba sisabhekwa njengomuthi wokuhlola. Amafutha awavunyelwe yi-US Food and Drug Administration (FDA) yokuthengisa. Lokho kusho ukuthi ezinye izibambiso zingase zihlanganise izindleko zokwelashwa kokuhlolwa. Amafutha ahlanganiswa ngokuhlanganyela yiCroda International yaseBrithani ne-SHS International. I-SHS International yi-distributor yomhlaba wonke wamafutha.

Izinsiza Ezihlobene Nokufunda Okuqhubekayo

Ufuna ukufunda okwengeziwe nge-ALD, i-Lorenzo Odone, nezinye izifo ezingavamile zofuzo?

Thola ama-athikili amaningi lapha:

Umthombo:

"Imininingwane Ngamafutha KaLornzo." I-Myelin Project. 13 Feb 2009