He aha ʻo Migalastat: Nā Hoʻohana, Ka Laʻau, Nā ʻAoʻao ʻAoʻao a me nā mea hou aku

He lāʻau lapaʻau ʻo Migalastat i kauoha ʻia no ka mālama ʻana i ka maʻi ʻo Fabry, he maʻi genetic kakaʻikahi e pili ana i ke ʻano o ka hana ʻana o kou kino i kekahi mau momona. Hana kēia lāʻau lapaʻau waha ma ke ʻano he "pharmacological chaperone," e kōkua ana i nā enzymes o kou kino e hana ʻoi aku ka maikaʻi ke hana pono ʻole lākou ma muli o nā loli genetic.

Inā ua ʻike ʻia ʻoe a i ʻole kekahi mea aloha me ka maʻi ʻo Fabry, hiki iā ʻoe ke manaʻo e lanakila ʻia e nā huaʻōlelo lapaʻau a me nā koho lapaʻau. ʻO ka hoʻomaopopo ʻana i ka hana ʻana o ka migalastat hiki ke kōkua iā ʻoe e manaʻoʻiʻo i kāu huakaʻi lapaʻau a me ka mea e manaʻo ai ma ke ala.

He aha ʻo Migalastat?

He lāʻau liʻiliʻi ʻo Migalastat e pili ana i kahi papa i kapa ʻia ʻo pharmacological chaperones. E noʻonoʻo iā ia he mea kōkua e kōkua ana i nā enzymes maoli o kou kino i ka hana ʻana i kā lākou hana me ka ʻoi aku ka maikaʻi.

ʻO ka mea kikoʻī, kōkua ʻo migalastat i ka hoʻopaʻa ʻana i kahi enzyme i kapa ʻia ʻo alpha-galactosidase A, ʻaʻole lawa a ʻaʻole hana pono i ka poʻe me ka maʻi ʻo Fabry. Ke hana pono ʻole kēia enzyme, e kūkulu ʻia kekahi mau mea momona i loko o kāu mau cell, e alakaʻi ana i nā hōʻailona o ka maʻi ʻo Fabry.

Hele mai ka lāʻau lapaʻau i ke ʻano capsule a lawe ʻia e ka waha. Ua kūʻai ʻia ma lalo o ka inoa inoa ʻo Galafold a ua ʻae ʻia e ka FDA i ka makahiki 2018 no ka mālama ʻana i nā pākeke me ka maʻi ʻo Fabry i hōʻoia ʻia.

No ke aha e hoʻohana ʻia ai ʻo Migalastat?

Hoʻohana ʻia ʻo Migalastat no ka mālama ʻana i ka maʻi ʻo Fabry i nā pākeke i loaʻa nā loli genetic kikoʻī e pane i kēia ʻano lapaʻau. He maʻi hoʻoilina kakaʻikahi ka maʻi ʻo Fabry e pili ana i nā ʻōkana he nui i kou kino.

Pono kāu kauka e hōʻoia ma o ka hoʻāʻo genetic e loaʻa iā ʻoe ke ʻano kūpono o ka loli maʻi ʻo Fabry ma mua o ke kau ʻana i ka migalastat. ʻAʻole nā ​​ʻano genetic a pau o ka maʻi ʻo Fabry e pane i kēia lāʻau lapaʻau, ʻo ia ke kumu e koʻikoʻi ai ka hoʻāʻo.

Kōkua ka lāʻau lapaʻau i ka hoʻēmi ʻana i ka hōʻiliʻili ʻana o nā mea momona i loko o kāu mau keena, hiki ke hoʻolohi a pale paha i kekahi mau pilikia e pili ana i ka maʻi ʻo Fabry. Hiki i kēia mau pilikia ke hoʻopilikia i kou puʻuwai, nā puʻupaʻa, ka ʻōnaehana nerve, a me ka ʻili.

Pehea e Hana ai ʻo Migalastat?

Hana ʻo Migalastat ma ka hoʻopaʻa ʻana a me ka hoʻopaʻa ʻana i ka enzyme alpha-galactosidase A i loko o kāu mau keena. Na kēia enzyme e wāwahi i kahi mea momona i kapa ʻia ʻo globotriaosylceramide (GL-3).

Ke loaʻa iā ʻoe ka maʻi ʻo Fabry, ʻaʻole hana kou kino i ka lawa o kēia enzyme a i ʻole hana i kahi mana i hana pono ʻole. Hana ʻo Migalastat e like me ka scaffold molecular, e kōkua ana i ka enzyme e mālama i kona ʻano kūpono a hana pono.

Manaʻo ʻia he lāʻau lapaʻau ikaika i ka hana kikoʻī, akā ua ʻae maikaʻi ʻia e ka hapa nui o nā kānaka. Pono ka lāʻau lapaʻau e hiki i kekahi mau pae i loko o kou kahe koko e pono ai, ʻo ia ke kumu he mea nui ka hoʻohana mau ʻana.

Pehea wau e lawe ai iā Migalastat?

E lawe iā migalastat e like me ke kauoha a kāu kauka, maʻamau hoʻokahi i kēlā me kēia lā ʻē aʻe i ka manawa like. ʻO ka maʻamau maʻamau he 123 mg, akā e hoʻoholo kāu kauka i ka nui kūpono no kāu kūlana.

Pono ʻoe e lawe iā migalastat ma ka ʻōpū ʻole, ma ka liʻiliʻi he 2 mau hola ma mua a i ʻole 2 mau hola ma hope o ka ʻai ʻana i kekahi meaʻai. He mea nui kēia no ka mea hiki i ka meaʻai ke keakea i ka maikaʻi o kou kino e komo ai i ka lāʻau lapaʻau.

E ale i ka capsule holoʻokoʻa me ke aniani wai. Mai ʻoki, nau, a wehe paha i ka capsule, no ka mea, hiki i kēia ke hoʻopilikia i ka hana ʻana o ka lāʻau lapaʻau. E hoʻāʻo e lawe i ka manawa like i kēlā me kēia lā e kōkua iā ʻoe e hoʻomanaʻo a mālama i nā pae kūlike i kou kino.

Inā ʻoe e lawe ana iā migalastat, e pale i ka inu ʻana i nā mea nui o ke kī, ke kofe, a i ʻole nā mea inu caffeinated ʻē aʻe i ka manawa e lawe ai ʻoe i kāu mahele, no ka mea, hiki i kēia mau mea ke keakea i ka komo ʻana.

Pehea ka lōʻihi e lawe ai au iā Migalastat?

He lāʻau lapaʻau lōʻihi ʻo Migalastat, pono ʻoe e lawe no ka manawa lōʻihi, a hiki paha i ke ola. No ka mea he maʻi genetic maʻi ʻo Fabry, pono ka mālama mau ʻana e mālama i nā pōmaikaʻi.

E nānā kāu kauka i kou pane ʻana i ka lāʻau lapaʻau ma o nā hoʻokolohua koko maʻamau a me nā loiloi ʻē aʻe. E nānā lākou i nā hōʻailona e hōʻike ana i ka maikaʻi o ka hana ʻana o ka lāʻau lapaʻau a inā e hoʻomaikaʻi ana a paʻa paha nā hōʻailona o kou maʻi Fabry.

Hiki ke ʻokoʻa ka manawa e ʻike ai i nā pōmaikaʻi mai kēlā kanaka i kēia kanaka. Hiki i kekahi poʻe ke ʻike i nā hoʻomaikaʻi ʻana i kekahi mau hōʻailona i loko o kekahi mau mahina, ʻoiai ʻo nā mea ʻē aʻe e lōʻihi ka manawa e ʻike ai i nā hopena piha o ka lāʻau lapaʻau.

He aha nā hopena ʻaoʻao o Migalastat?

E like me nā lāʻau lapaʻau a pau, hiki i ka migalastat ke hana i nā hopena ʻaoʻao, ʻoiai ʻaʻole e ʻike nā mea a pau iā lākou. ʻO ka hapa nui o nā hopena ʻaoʻao he maʻalahi a i ka waena a maʻa mau e hoʻomaikaʻi i ka wā e hoʻoponopono ai kou kino i ka lāʻau lapaʻau.

ʻO nā hopena ʻaoʻao maʻamau i ʻike ʻia e nā kānaka, ʻo ia ka ʻehaʻeha, ka nausea, a me nā maʻi maʻi urinary tract. Hiki ke hana pinepine kēia i nā pule mua o ka lāʻau lapaʻau a pinepine e lilo i mea liʻiliʻi i ka manawa.

Eia nā hopena ʻaoʻao āu e ʻike ai, i hui pū ʻia e ka maʻamau o lākou:

Nā hopena ʻaoʻao maʻamau (e pili ana i ka nui ma mua o 1 i 10 mau kānaka):

• ʻEhaʻeha
• Nausea
• Nā maʻi urinary tract
• Nā maʻi maʻi hanu luna

Nā hopena ʻaoʻao liʻiliʻi (e pili ana i 1 i 10 a 1 i 100 mau kānaka):

• ʻĀwili
• Luhi
• Pāpaʻa
• Paʻa
• ʻEha o ka ʻōpū
• Nā spasms ʻiʻo
• ʻEha hope

Nā hopena ʻaoʻao kakaʻikahi (e pili ana i ka liʻiliʻi ma mua o 1 i 100 mau kānaka):

• Nā hopena maʻi maʻi koʻikoʻi
• Nā loli nui i ka hana o ka puʻupaʻa
• Nā loli maʻamau i ka puʻuwai
• Nā pilikia digestive koʻikoʻi

He nui ka poʻe e ʻae maikaʻi ana iā migalastat, a ʻaʻole maʻamau nā hopena ʻaoʻao koʻikoʻi. Eia naʻe, he mea nui e hōʻike i nā hōʻailona e hopohopo nei i kāu mea mālama ola, ʻoiai inā e hoʻomau a ʻino paha lākou i ka manawa.

ʻO wai ka mea pono ʻole e lawe iā Migalastat?

ʻAʻole kūpono ʻo Migalastat no nā mea a pau i loaʻa i ka maʻi Fabry. Pono kāu kauka e hōʻoia ma o ka hoʻāʻo genetic e loaʻa iā ʻoe nā loli kūpono ma mua o ke kau ʻana i kēia lāʻau.

ʻAʻole pono ʻoe e lawe iā migalastat inā he maʻi ʻoe i ka lāʻau a i ʻole kekahi o kāna meaʻai. E haʻi i kāu kauka e pili ana i nā hopena maʻi āu i loaʻa ai i nā lāʻau.

Pono paha ka poʻe me ka maʻi kīkī koʻikoʻi i ka nānā ʻana a i ʻole nā hoʻoponopono ʻana i ka lāʻau, no ka mea, ua hana ʻia ka lāʻau ma o nā kīkī. E nānā kāu kauka i kāu hana kīkī maʻamau inā e lawe ana ʻoe iā migalastat.

Inā hāpai ʻoe a i ʻole e hoʻolālā ana e hāpai, e kūkākūkā i kēia me kāu kauka. ʻOiai he palena ka ʻikepili e pili ana i ka hoʻohana ʻana iā migalastat i ka wā hāpai, e kaupaona kāu kauka i nā pōmaikaʻi kūpono e kūʻē i nā pilikia.

Nā inoa inoa o Migalastat

Hoʻolaha nui ʻia ʻo Migalastat ma lalo o ka inoa inoa ʻo Galafold. ʻO kēia ka inoa maʻamau āu e ʻike ai ma nā palapala kauoha a me nā hue lāʻau.

Hana ʻia ka lāʻau e Amicus Therapeutics a loaʻa ma ka hapa nui o nā ʻāina kahi i loaʻa ai iā ia ka ʻae hoʻoponopono. ʻAʻole i loaʻa nā mana generic o migalastat i kēia manawa, no ka mea, aia nō ka lāʻau ma lalo o ka pale palapala.

Ke kūkākūkā ʻana i kāu mālama ʻana me nā mea mālama ola a i ʻole nā ​​kānaka lapaʻau, hiki iā ʻoe ke kuhikuhi iā ia ma kekahi inoa - migalastat a i ʻole Galafold - a e maopopo iā lākou ke ʻano o ka lāʻau āu e kamaʻilio nei.

Nā koho ʻē aʻe o Migalastat

No ka poʻe me ka maʻi Fabry, ʻo ka mea ʻē aʻe nui i ka migalastat ʻo ka therapy pani enzyme (ERT) me ka hoʻohana ʻana i nā lāʻau e like me agalsidase alfa a i ʻole agalsidase beta. Hāʻawi ʻia kēia mau mea ma ke ʻano he intravenous infusions ma mua o nā capsules waha.

A koho ma waena o migalastat a me ERT e hilinaʻi ana i nā mea he nui, me kāu hoʻololi genetic kikoʻī, pehea ka maikaʻi o kou ʻae ʻana i kēlā me kēia lapaʻau, a me kāu mau makemake pilikino e pili ana i nā ʻano lapaʻau.

Hiki i kekahi poʻe ke hoʻololi ma waena o nā lapaʻau a i ʻole e hoʻohana iā lākou i ka hui pū ʻana, ma muli o kā lākou pane a me nā hopena ʻaoʻao a lākou e ʻike ai. E kōkua kāu kauka e hoʻoholo i ke ala maikaʻi loa no kou kūlana pilikino.

Aia kekahi noiʻi e hana nei i nā lapaʻau ʻē aʻe no ka maʻi ʻo Fabry, me ka gene therapy a me nā ala hou ʻē aʻe, ʻoiai aia nō kēia mau mea i nā pae hoʻokolohua.

Ua ʻoi aku ka maikaʻi o Migalastat ma mua o ka Enzyme Replacement Therapy?

Inā ʻoi aku ka maikaʻi o ka migalastat ma mua o ka enzyme replacement therapy e hilinaʻi ana i kou kūlana pilikino a me ke ʻano genetic. Hiki i nā lapaʻau ʻelua ke maikaʻi no ka mālama ʻana i ka maʻi ʻo Fabry, akā hana lākou ma nā ʻano like ʻole.

Hāʻawi ʻo Migalastat i ka maʻalahi o ka hoʻohana ʻana i ka waha i kēlā me kēia lā ʻē aʻe, ʻoiai ke koi ʻia ka enzyme replacement therapy i nā infusions intravenous i kēlā me kēia pule ʻelua. Nui ka poʻe makemake i ka maʻalahi o ka lawe ʻana i ka pila ma ka home ma mua o ka hele ʻana i kahi keʻena mālama ola kino no nā infusions.

Eia naʻe, hana wale ʻo migalastat no ka poʻe me nā hoʻololi genetic kikoʻī i

Hiki ke hoʻohana palekana ʻia ʻo Migalastat i ka poʻe me ka maʻi puʻuwai, me ka poʻe me nā pilikia o ka puʻuwai e pili ana i ka maʻi Fabry. ʻO ka ʻoiaʻiʻo, ʻo ka mālama ʻana i ka maʻi Fabry me migalastat hiki ke kōkua i ka hoʻomaikaʻi ʻana i kekahi mau hōʻailona e pili ana i ka puʻuwai i ka wā.

Eia naʻe, makemake kāu kauka e nānā mau i ka hana o kou puʻuwai i ka wā e lawe ana ʻoe i ka migalastat. Hiki iā lākou ke ʻōlelo aku i nā hoʻokolohua puʻuwai manawa e like me nā echocardiograms a i ʻole nā ​​electrocardiograms e hōʻoia ʻaʻole e hoʻokumu ka lāʻau i kekahi pilikia.

Inā loaʻa iā ʻoe ka maʻi puʻuwai koʻikoʻi a i ʻole i loaʻa i nā pilikia o ka puʻuwai i kēia manawa, pono paha kāu kauka e hoʻoponopono i kāu hoʻolālā lapaʻau a i ʻole e nānā pono iā ʻoe i ka wā e hoʻomaka ai ʻoe i ka migalastat.

He aha kaʻu e hana ai inā lawe hewa au i ka nui o Migalastat?

Inā lawe hewa ʻoe i ka nui o ka migalastat ma mua o ka mea i kauoha ʻia, e hoʻokaʻaʻike koke i kāu kauka a i ʻole ke kikowaena mana ʻawaʻawa. Mai kali e ʻike inā e ulu nā hōʻailona, ​​no ka mea, he mea nui ka loaʻa ʻana o ka ʻōlelo aʻo lapaʻau wikiwiki.

ʻOiai ʻo ka lawe ʻana i nā mea hou aku i kekahi manawa ʻaʻole paha e hōʻeha nui, ʻaʻole ia i ʻōlelo ʻia a hiki ke hoʻonui i kou pilikia o nā hopena ʻaoʻao. ʻO nā hōʻailona o ka lawe ʻana i ka nui e komo paha i ka piʻi ʻana o ka nausea, ke poʻo, a i ʻole nā ​​pilikia digestive.

E mālama i ka hue lāʻau me ʻoe i ka wā e ʻimi ai i ka lāʻau lapaʻau, no ka mea, e kōkua kēia i nā mea mālama ola e hoʻomaopopo pono i ka mea a me ka nui āu i lawe ai. A laila hiki iā lākou ke hāʻawi i ka mālama kūpono loa e pili ana i kāu kūlana.

He aha kaʻu e hana ai inā haʻalele au i kahi mahele o Migalastat?

Inā haʻalele ʻoe i kahi mahele o ka migalastat, e lawe koke iā ia ke hoʻomanaʻo ʻoe, inā aia i loko o 12 mau hola o kou manawa dosing maʻamau. Inā ua hala ma mua o 12 mau hola, e lele i ka mahele i haʻalele ʻia a lawe i kāu mahele aʻe i ka manawa maʻamau.

Mai lawe i ʻelua mahele kokoke i kekahi i kekahi e hana i kahi mahele i haʻalele ʻia, no ka mea, hiki i kēia ke hoʻonui i kou pilikia o nā hopena ʻaoʻao. Akā, e hoʻomau i kāu papa hana maʻamau i kēlā me kēia lā.

Inā poina pinepine ʻoe i nā lāʻau, e noʻonoʻo e hoʻonohonoho i kahi hoʻomanaʻo kelepona a i ʻole e hoʻohana i kahi mea hoʻonohonoho pila e kōkua iā ʻoe e hoʻomanaʻo. He mea nui ka lawe ʻana i nā lāʻau i nā manawa kūpono no ka mālama ʻana i ka pono o ka lāʻau lapaʻau i ka mālama ʻana i kou maʻi Fabry.

I ka manawa hea e hiki ai iaʻu ke hooki i ka lawe ʻana iā Migalastat?

Pono ʻoe e hooki i ka lawe ʻana iā migalastat ma lalo o ka mālama ʻana a kāu kauka. No ka mea he maʻi genetic ola mau ka maʻi Fabry, hiki i ka hooki ʻana i ka lāʻau lapaʻau ke ʻae i nā hōʻailona e hoʻi mai a i ʻole e ʻino aʻe i ka manawa.

Hiki i kāu kauka ke ʻōlelo e hooki i ka migalastat inā loaʻa iā ʻoe nā hopena ʻaoʻao koʻikoʻi ʻaʻole hiki ke mālama ʻia, inā ʻaʻole hana pono ka lāʻau lapaʻau iā ʻoe, a i ʻole inā loaʻa kahi koho lapaʻau maikaʻi aʻe.

Inā ʻoe e noʻonoʻo ana e hooki i ka lāʻau lapaʻau ma muli o nā hopena ʻaoʻao a i ʻole nā hopohopo ʻē aʻe, e kamaʻilio mua me kāu kauka. Hiki iā lākou ke hoʻoponopono i kāu hoʻolālā lapaʻau a i ʻole e hāʻawi i nā hoʻolālā e kōkua iā ʻoe e hoʻomau i ka lāʻau lapaʻau me ka palekana.

Hiki iaʻu ke lawe iā Migalastat me nā lāʻau lapaʻau ʻē aʻe?

Hiki i ka Migalastat ke launa pū me kekahi mau lāʻau lapaʻau, no laila he mea nui e haʻi i kāu kauka e pili ana i nā lāʻau lapaʻau a pau, nā mea hoʻohui, a me nā lāʻau lapaʻau i kūʻai ʻia me ka ʻole o ka palapala kauoha āu e lawe nei. Pākuʻi ʻia kēia i nā huaora, nā mea hoʻohui lāʻau, a me nā lāʻau lapaʻau āu e kūʻai ai me ka ʻole o ka palapala kauoha.

ʻO kekahi mau lāʻau lapaʻau e launa pū me ka migalastat e pili ana i kekahi mau antacids, nā lāʻau lapaʻau e hōʻemi ana i ka waikawa, a me kekahi mau antibiotics. Hiki i kāu kauka a i ʻole ka mea kūʻai lāʻau lapaʻau ke nānā i nā pilina hiki ke hana a hoʻoponopono i kāu hoʻolālā lapaʻau inā pono.

Inā pono ʻoe e hoʻomaka i kahi lāʻau lapaʻau hou i ka wā e lawe ana iā migalastat, e hōʻoia i ka ʻike o kāu mau mea mālama ola kino a pau e pili ana i kāu lapaʻau maʻi Fabry. Kōkua kēia i ka hōʻoia ʻana e hiki iā lākou ke kauoha i nā lāʻau lapaʻau ʻaʻole e hoʻopilikia i kāu lāʻau lapaʻau migalastat.