He aha ka Onasemnogene Abeparvovec (Zolgensma): Nā Hoʻohana, Ka Leka, Nā ʻAoʻao ʻAoʻao a me nā mea hou aku

ʻO Onasemnogene abeparvovec, i ʻike ʻia e kona inoa inoa ʻo Zolgensma, he lāʻau lapaʻau gene i hoʻolālā ʻia e mālama i ka atrophy muscular spinal (SMA) i nā keiki ʻōpio. Hana kēia lapaʻau hoʻokahi manawa ma ka hāʻawi ʻana i kahi kope olakino o ka gene SMN1 pololei i nā neurons motika o ke keiki, e kōkua ana i ko lākou kino e hana i ka protein e pono ai no ka hana ʻana o ka ʻiʻo.

Inā ua ʻike ʻia kāu keiki me SMA, e manaʻo ana paha ʻoe i ka nui o nā nīnau a me nā hopohopo. Hōʻike kēia lāʻau lapaʻau hou i ka manaʻolana no nā ʻohana e kū nei i kēia kūlana paʻakikī, e hāʻawi ana i ka hiki ke hoʻolohi a i ʻole ke kāpae ʻana i ka holomua o ka nāwaliwali o ka ʻiʻo e hōʻike ana iā SMA.

He aha ka Onasemnogene Abeparvovec?

ʻO Onasemnogene abeparvovec kahi lāʻau lapaʻau gene e pani ai i ka gene SMN1 nalowale a hewa paha i nā keiki me ka atrophy muscular spinal. ʻAʻole like me nā lāʻau lapaʻau kuʻuna āu e lawe pinepine ai, hāʻawi ʻia kēia lapaʻau ma ke ʻano he infusion intravenous hoʻokahi e manaʻo ana e hāʻawi i nā pōmaikaʻi mau.

Hoʻohana ka lāʻau lapaʻau i kahi maʻi i hoʻololi ʻia i kapa ʻia ʻo adeno-associated virus (AAV) ma ke ʻano he ʻōnaehana hāʻawi. E noʻonoʻo i kēia maʻi ma ke ʻano he ʻelele kōkua e lawe ana i ka gene olakino pololei i nā neurons motika i loko o ke kuamoʻo o kāu keiki. ʻO kēia mau neurons motika nā pūnaewele e mālama ana i ka neʻe ʻana o ka ʻiʻo, a ma SMA, ʻaʻole lākou e hana pono ma muli o ka gene nalowale a hemahema paha.

Ua ʻae ʻia kēia lāʻau lapaʻau e ka FDA i ka makahiki 2019 a hōʻike i ka lāʻau lapaʻau gene mua no SMA. Ua hoʻolālā kūikawā ʻia no nā keiki ma lalo o 2 mau makahiki, no ka mea, i kēia manawa e hana maikaʻi loa ka lāʻau lapaʻau.

No ke aha e hoʻohana ʻia ai ka Onasemnogene Abeparvovec?

Hoʻohana ʻia ka Onasemnogene abeparvovec e mālama i ka atrophy muscular spinal (SMA) i nā keiki ma lalo o 2 mau makahiki. ʻO SMA kahi maʻi genetic e pili ana i nā neurons motika, e alakaʻi ana i ka nāwaliwali o ka ʻiʻo a me ka nalowale o nā hiki ke neʻe.

He mea pono loa ka lāʻau lapaʻau no nā keiki me SMA Type 1, ke ʻano koʻikoʻi loa o ke kūlana. Hōʻike maʻamau nā keiki me SMA Type 1 i nā hōʻailona i loko o nā mahina mua o ke ola, me ka pilikia o ka hanu ʻana, ka ʻai ʻana, a me ka paʻa ʻana i ko lākou poʻo. Me ka ʻole o ka lāʻau lapaʻau, nui nā keiki me kēia ʻano ʻaʻole e ola ma mua o ko lākou lā hānau ʻelua.

Hiki i kāu kauka ke noʻonoʻo i kēia lāʻau lapaʻau no nā keiki me SMA Type 2 a i ʻole nā ​​mea i hōʻike ʻole i nā hōʻailona akā ua loaʻa nā hoʻokolohua genetic e hōʻike ana e hoʻomohala lākou i ka SMA. ʻO ke kī ka hana mua, no ka mea, hana maikaʻi ka lāʻau lapaʻau ke hāʻawi ʻia ma mua o ka pōʻino nui o ka neuron motor.

Pehea e hana ai ʻo Onasemnogene Abeparvovec?

Hana ʻo Onasemnogene abeparvovec ma ka hāʻawi ʻana i kahi kope hana o ka gene SMN1 i nā neurons motor o kāu keiki. Manaʻo ʻia he lāʻau lapaʻau ikaika a hiki ke hoʻololi i ke ola no ka mea e pili ana i ke kumu kumu o SMA ma mua o ka mālama ʻana i nā hōʻailona.

I nā kānaka olakino, hana ka gene SMN1 i kahi protein i kapa ʻia ʻo ka protein survival motor neuron (SMN), he mea nui ia no ka hana neuron motor. ʻAʻole loaʻa i nā keiki me SMA kēia gene a i ʻole he mana hemahema ʻaʻole e hana i ka protein hana lawa.

Hoʻohana ka lāʻau lapaʻau i kahi maʻi i hoʻololi ʻia e lawe i ka gene olakino pololei i nā neurons motor i loko o ke kuamoʻo. I laila, hoʻomaka ka gene e hana i ka protein SMN e pono nui ai kēia mau cell. Hiki i kēia kaʻina hana ke kōkua i ka pale ʻana i ka make hou ʻana o ka neuron motor a hiki ke kōkua i ka hoʻihoʻi ʻana i kekahi hana i nā cell i pōʻino.

Hoʻolālā ʻia ka lāʻau lapaʻau e hāʻawi i nā pōmaikaʻi lōʻihi mai kahi mahele hoʻokahi, ʻoiai hiki ke ʻokoʻa ka nui o ka hoʻomaikaʻi ʻana mai ke keiki a ke keiki. Hiki i kekahi mau keiki ke ʻike i ka paʻa ʻana o ko lākou kūlana, ʻoiai e hōʻike ana kekahi i nā hoʻomaikaʻi ʻana i ka hana motor i ka manawa.

Pehea wau e hoʻomākaukau ai no ka lāʻau lapaʻau Onasemnogene Abeparvovec?

Ma mua o ka loaʻa ʻana o kāu keiki i ka onasemnogene abeparvovec, e hana kāu hui lapaʻau i nā hoʻomākaukau piha e hōʻoia i ka palekana a me ka pono o ka lapaʻau. ʻAʻole kēia he mea e pono ai ʻoe e hele wale - e alakaʻi kāu hui mālama ola iā ʻoe ma kēlā me kēia ʻanuʻu.

Pono kāu keiki i nā hoʻokolohua piha ma mua o ka lapaʻau ʻana, me ka hana koko e nānā i ka hana o ke ake a me ke kūlana o ka ʻōnaehana pale. E loiloi pū ka hui lapaʻau i ke olakino holoʻokoʻa o kāu keiki a me ka hana hanu, no ka mea, pono paha kekahi mau keiki i ke kākoʻo hanu i ka wā a ma hope o ka lapaʻau.

Eia ka mea e hiki iā ʻoe ke manaʻo i ka wā o ka hoʻomākaukau ʻana:

• Nā lāʻau lapaʻau ma mua o ka lapaʻau ʻana e hōʻemi i ka pilikia o nā hopena pale
• ʻO ka lāʻau corticosteroid e hoʻomaka ana ma mua o ka hoʻokomo ʻana a e hoʻomau ana no kekahi mau pule
• Nā kūkākūkā kikoʻī e pili ana i ka mea e manaʻo ai i ka wā a ma hope o ka lapaʻau
• Hoʻolālā no ka nānā ʻana ma hope o ka lapaʻau a me ka mālama ʻana
• Hoʻopili pū me nā loea e komo i ka mālama mau ʻana o kāu keiki

Hāʻawi ʻia ka hoʻokomo ʻana i loko o kahi haukapila ma kahi o 60 mau minuke. E nānā pono ʻia kāu keiki i ke kaʻina hana, a hiki iā ʻoe ke noho pū me lākou i ka wā o ka lapaʻau.

Pehea ka lōʻihi o ka lapaʻau ʻana o Onasemnogene Abeparvovec?

Hoʻolālā ʻia ka Onasemnogene abeparvovec ma ke ʻano he lapaʻau hoʻokahi manawa e hiki ke hāʻawi i nā pōmaikaʻi mau i ke ola o kāu keiki. ʻAʻole like me nā lāʻau lapaʻau e pono ai e lawe ʻia i kēlā me kēia lā a i ʻole i kēlā me kēia pule, manaʻo kēia gene therapy e hana i nā loli mau i nā neurons kaʻa o kāu keiki.

Hana ka lapaʻau ma ka hoʻohui ʻana i ka gene SMN1 olakino i loko o nā neurons kaʻa o kāu keiki, kahi e hoʻomau ai i ka hana ʻana i ka protein e pono ai no nā makahiki e hiki mai ana. Hōʻike ka noiʻi e hiki ke lōʻihi nā pōmaikaʻi, ʻoiai e aʻo ana nā ʻepekema i nā hopena lōʻihi mai ka mea hou o ka lapaʻau.

Eia naʻe, pono kāu keiki i ka nānā mau ʻana a me ka mālama kākoʻo ʻana ma hope o ka loaʻa ʻana o ka gene therapy. Pākuʻi ʻia kēia i nā nānā maʻamau e loiloi i ka hana kaʻa, ka hanu ʻana, a me ka ulu holoʻokoʻa. Pono paha kekahi mau keiki i ka physical therapy, occupational therapy, a i ʻole nā lāʻau lapaʻau kākoʻo ʻē aʻe e kōkua iā lākou e hiki i ko lākou hiki piha.

He aha nā hopena ʻaoʻao o Onasemnogene Abeparvovec?

E like me nā lāʻau lapaʻau ikaika, hiki i ka onasemnogene abeparvovec ke hana i nā hopena ʻaoʻao, akā nui nā keiki e ʻae maikaʻi i ka lāʻau lapaʻau. ʻO ka hoʻomaopopo ʻana i kēia mau hopena hiki ke kōkua iā ʻoe e mākaukau a ʻike i ka mea e nānā ai.

ʻO nā hopena ʻaoʻao maʻamau ka mea e mālama ʻia a hoʻoponopono pinepine ʻia me ka mālama olakino kūpono. E nānā pono kāu hui mālama ola i kāu keiki a hāʻawi i nā lāʻau lapaʻau e hōʻemi i nā pilikia.

ʻO nā hopena ʻaoʻao maʻamau āu e ʻike ai:

• Hoʻonui ʻia nā enzymes ate, e nānā ai kāu kauka ma o nā hoʻokolohua koko
• Luaʻi a i ʻole ka emi ʻana o ka ʻai i nā lā ma hope o ka lāʻau lapaʻau
• Fiva a i ʻole ka huhū i ka wā e pane ai ka ʻōnaehana pale o kāu keiki i ka lāʻau lapaʻau
• Hoʻonui i ka hiamoe a i ʻole nā loli i nā pae hana maʻamau
• Nā hōʻailona hanu māmā a i ʻole nā loli i nā ʻano hanu

Hiki i nā hopena ʻaoʻao koʻikoʻi akā ʻaʻole maʻamau ke komo i nā pilikia ate nui a i ʻole nā hopena immune koʻikoʻi. E nānā pono kāu hui lapaʻau i kēia mau mea a hana koke inā pono.

ʻO nā hopena ʻaoʻao laha akā koʻikoʻi e koi ana i ka lāʻau lapaʻau koke:

• ʻO ka hana ʻino ʻana o ka ate, hiki ke hana i ka melemele o ka ʻili a i ʻole nā maka
• Nā hāʻule nui i ka helu platelet, e alakaʻi ana i ka ʻeha a i ʻole ke kahe ʻana
• Nā hopena maʻi maʻi koʻikoʻi i ka wā a i ʻole ma hope koke o ka infusion
• Nā pilikia hanu koʻikoʻi a i ʻole ka pilikia hanu
• Nā hōʻailona o nā pilikia o ka naʻau, e like me ka puʻuwai ʻole a i ʻole ka ʻeha o ka umauma

E hoʻomanaʻo i ka ʻike o kāu hui mālama ola i ka mālama ʻana i kēia mau hopena ʻaoʻao a e loaʻa nā hoʻolālā e hoʻoponopono i nā pilikia e kū mai ana.

ʻO wai ka mea pono ʻole e lawe i ka Onasemnogene Abeparvovec?

ʻOiai ke hāʻawi nei ka onasemnogene abeparvovec i ka manaʻolana no nā keiki he nui me SMA, ʻaʻole kūpono ia no nā mea a pau. E loiloi pono kāu kauka inā kūpono kēia lapaʻau no kāu keiki ma muli o kekahi mau mea nui.

ʻO nā keiki ma mua o 2 mau makahiki ʻaʻole e loaʻa kēia lapaʻau, no ka mea, ʻoi aku ka maikaʻi ke hāʻawi ʻia i ka wā ʻōpiopio. ʻOi aku ka maikaʻi o ka therapy ke olakino nā neurons kaʻa a ʻaʻole i loaʻa ka pōʻino nui.

ʻAʻole paha he moho kāu keiki no kēia lapaʻau inā loaʻa iā lākou:

• Maʻi ate koʻikoʻi a i ʻole nā ​​enzyme ate i hoʻokiʻekiʻe nui ʻia
• SMA holomua me ka nalowale nui o ka neuron kaʻa
• Nā maʻi puʻuwai koʻikoʻi e hoʻoweliweli ai i ka infusion
• Nā pilikia ʻōnaehana immune koʻikoʻi e hiki ke hoʻopilikia i ka lapaʻau
• ʻO ka ʻike mua ʻana i ka AAV i hana i nā antibodies e kūʻē i ka maʻi

ʻO nā mea i manaʻo ʻia akā he mea nui:

• Nā maʻi maʻi e pono e mālama ʻia ma mua o ka gene therapy
• Nā pilikia hanu koʻikoʻi e hoʻoweliweli ai i ka infusion
• Nā kūlana genetic ʻē aʻe e hiki ke hoʻopilikia i ka lapaʻau
• Nā hopena maʻi maʻi koʻikoʻi i nā lapaʻau like

E kūkākūkā kāu hui lapaʻau i kēia mau mea a pau me ʻoe a kōkua i ka hoʻoholo ʻana i ke ala lapaʻau maikaʻi loa no ke kūlana kikoʻī o kāu keiki.

Inoa Brand Onasemnogene Abeparvovec

Hoʻolaha ʻia ka Onasemnogene abeparvovec ma lalo o ka inoa brand Zolgensma. ʻO kēia inoa ka mea āu e lohe pinepine ai i nā kūkākūkā me kāu hui mālama ola a me nā palapala lapaʻau.

Hana ʻia ʻo Zolgensma e Novartis Gene Therapies a ua hana kūikawā ʻia no ka mālama ʻana i ka SMA i nā keiki ʻōpio. ʻOi aku ka maʻalahi o ka inoa inoa e hoʻomanaʻo a hoʻopuka ma mua o ka inoa generic piha, no laila, mai hopohopo inā ʻoe e hoʻohana pinepine iā "Zolgensma" i nā kamaʻilio.

Ke kūkākūkā ʻana i nā koho lapaʻau a i ʻole ka uhi ʻinikua, hiki iā ʻoe ke lohe i nā inoa ʻelua i hoʻohana ʻia me ka hoʻololi ʻana. E pili ana lākou i ka lāʻau lapaʻau like, no laila ʻaʻohe ʻokoʻa i ka lapaʻau āu e loaʻa nei me ka nānā ʻole i ka inoa i hoʻohana ʻia.

Nā Koho ʻē aʻe o Onasemnogene Abeparvovec

ʻOiai he lapaʻau hoʻomaka ʻo onasemnogene abeparvovec, aia nā lāʻau lapaʻau ʻē aʻe i ʻae ʻia e ka FDA no SMA e noʻonoʻo ai kāu kauka. Hana ʻokoʻa kēlā me kēia lapaʻau a loaʻa nā pōmaikaʻi a me nā manaʻo ponoʻī.

ʻO Nusinersen (Spinraza) kekahi lāʻau lapaʻau i hoʻohana ʻia no ka mālama ʻana i ka SMA i nā keiki a me nā mākua. ʻAʻole like me ke ala lapaʻau gene hoʻokahi manawa, hāʻawi ʻia ʻo nusinersen ma ke ʻano he mau injections maʻamau i loko o ka wai spinal i kēlā me kēia mau mahina. Kōkua kēia lapaʻau i ke kino e hana i ka protein SMN hou mai ka gene SMN2 backup.

ʻO Risdiplam (Evrysdi) kahi lāʻau lapaʻau waha e hiki ke hāʻawi ʻia i nā keiki a me nā mākua me SMA. He wai ia e lawe ʻia i kēlā me kēia lā ma ka home, e maʻalahi ai no kekahi mau ʻohana. Hana pū kēia lāʻau lapaʻau ma ke kōkua ʻana i ka gene SMN2 e hana i ka protein hana hou.

E kōkua kāu kauka iā ʻoe e hoʻomaopopo i ka lapaʻau e kūpono loa no kāu keiki e pili ana i ko lākou makahiki, ke ʻano o SMA, nā hōʻailona o kēia manawa, a me nā mea olakino ʻē aʻe. I kekahi manawa, hoʻohana ʻia nā lapaʻau kākoʻo e like me ka lāʻau kino, ke kōkua hanu, a me ke kākoʻo meaʻai me kēia mau lāʻau lapaʻau.

ʻOi aku ka maikaʻi o Onasemnogene Abeparvovec ma mua o Nusinersen?

ʻAʻole maʻalahi ka hoʻohālikelike ʻana iā onasemnogene abeparvovec me nusinersen no ka mea he hana like ʻole lākou a kūpono no nā kūlana like ʻole. Ua hōʻike nā lāʻau lapaʻau ʻelua i nā pōmaikaʻi nui no nā keiki me SMA, akā ʻo ke koho maikaʻi loa e hilinaʻi ana i nā kūlana kikoʻī o kāu keiki.

Hāʻawi ʻo Onasemnogene abeparvovec i ka pōmaikaʻi o ka lapaʻau hoʻokahi e hoʻoponopono ana i ke kumu kumu o SMA ma ka hāʻawi ʻana i kahi kope olakino o ka gene nalowale. Hiki i kēia ke ʻoluʻolu loa no nā ʻohana e makemake e pale i nā kaʻina hana lapaʻau hou a me nā papa hana lapaʻau e hoʻomau nei.

ʻO Nusinersen, ma kekahi ʻaoʻao, hiki ke hoʻohana ʻia i nā keiki o nā makahiki āpau a he moʻolelo lōʻihi o ka palekana a me ka pono. Makemake kekahi mau ʻohana i ka maʻalahi o ka hiki ke hooki i ka lapaʻau inā pono, a ʻo ka nānā mau ʻana e hele pū me nā injections e hoʻomau nei hiki ke hāʻawi i ka hōʻoluʻolu.

E noʻonoʻo kāu hui mālama ola i nā mea e like me ka makahiki o kāu keiki, ka paʻakikī o nā hōʻailona, ​​ke olakino holoʻokoʻa, a me nā makemake o kou ʻohana i ka wā e ʻōlelo ai i ka lāʻau lapaʻau kūpono loa. I kekahi manawa, hiki mai ka hoʻoholo i nā mea e like me ka manawa, ka loaʻa, a me ka uhi ʻinikua.

Nā nīnau i nīnau pinepine ʻia e pili ana iā Onasemnogene Abeparvovec

He palekana anei ʻo Onasemnogene Abeparvovec no nā keiki hānau hou?

ʻAe, hiki ke hāʻawi palekana ʻia ʻo onasemnogene abeparvovec i nā keiki hānau hou a me nā pēpē ʻōpio loa ke hāʻawi ʻia e nā hui lapaʻau i ʻike ʻia. ʻO ka ʻoiaʻiʻo, ʻoi aku ka maikaʻi o ka lapaʻau ke hāʻawi ʻia i ka wā mua o ke ola, i kekahi manawa i loko o nā mahina mua ma hope o ka hānau ʻana.

Pono nā keiki hānau hou i ka nānā kūikawā i ka wā a ma hope o ka lapaʻau ʻana no ka mea e hoʻomohala ana kā lākou ʻōnaehana pale a me nā hana o nā ʻōpū. E mālama kāu hui lapaʻau i nā hana hou e hōʻoia i ka loaʻa ʻana o kāu pēpē i ka mālama palekana loa, me ka nānā pono ʻana i ka hana o ke ake a me ka hanu.

E hilinaʻi ka hoʻoholo e mālama i kahi keiki hānau hou i ko lākou olakino holoʻokoʻa, ka paʻakikī o kā lākou SMA, a me ko lākou hiki ke ʻae i ka lāʻau lapaʻau. E hana pū kāu mau kauka me ʻoe e hoʻoholo i ka manawa kūpono no ka lāʻau lapaʻau.

He aha kaʻu e hana ai inā hōʻike kaʻu keiki i nā hōʻailona e pili ana ma hope o ka lāʻau lapaʻau?

Inā ʻike ʻoe i nā hōʻailona e pili ana ma hope o ka loaʻa ʻana o kāu keiki i ka onasemnogene abeparvovec, e hoʻokaʻaʻike koke i kāu hui mālama ola. ʻOi aku ka maikaʻi o ka hana ʻana ma ka ʻaoʻao o ka akahele i ka wā e pili ana i ke olakino o kāu keiki.

ʻO nā hōʻailona e koi ai i ka lāʻau lapaʻau koke ʻana, ʻo ia ka melemele o ka ʻili a i ʻole nā maka, ka ʻeha a i ʻole ke kahe ʻana o ke koko, ka luaʻi nui, ka pilikia o ka hanu ʻana, a i ʻole nā hōʻailona o ka hopena maʻi. E hāʻawi kāu hui lapaʻau iā ʻoe i nā ʻōlelo kikoʻī e pili ana i ka mea e nānā ai a i ka wā e kāhea ai.

E hoʻomanaʻo i kekahi mau hopena ʻaoʻao maʻalahi he mea maʻamau a manaʻo ʻia ma hope o ka lāʻau lapaʻau. E kōkua kāu hui mālama ola iā ʻoe e hoʻokaʻawale ma waena o nā hopena i manaʻo ʻia a me nā hōʻailona e pono ai ka nānā koke.

Pehea ka wikiwiki e ʻike ai au i nā hoʻomaikaʻi ma hope o ka lāʻau lapaʻau?

ʻO ka manawa no ka ʻike ʻana i nā hoʻomaikaʻi ma hope o ka lāʻau lapaʻau onasemnogene abeparvovec e ʻokoʻa mai keiki a keiki. ʻIke kekahi mau ʻohana i nā loli i loko o kekahi mau pule, aʻo nā mea ʻē aʻe e ʻike i nā hoʻomaikaʻi mālie i nā mahina he nui.

He mea nui e hoʻomanaʻo e hana ana kēia lāʻau lapaʻau ma ka pale ʻana i ka nalowale ʻana o ka neuron motor a me ka hoʻomaikaʻi ʻana i ka hana i ka manawa. ʻO nā pōmaikaʻi e pili ana i ka mālama ʻana i nā hiki o kēia manawa a i ʻole ka hoʻolohi ʻana i ka emi ʻana ma mua o nā hoʻomaikaʻi koke.

E nānā kāu hui mālama ola i ka holomua o kāu keiki ma o nā loiloi maʻamau a hiki ke kōkua iā ʻoe e hoʻomaopopo i nā loli e manaʻo ai a i ka manawa. He mea kū hoʻokahi ka pane a kēlā me kēia keiki, a pono pinepine ke ahonui e ʻike i nā pōmaikaʻi piha o ka lāʻau lapaʻau.

I ka manawa hea mākou e noʻonoʻo ai i kēia lāʻau lapaʻau?

ʻO ka manawa maikaʻi loa e noʻonoʻo ai i ka onasemnogene abeparvovec i ka wā hiki ke hiki ma hope o ka hōʻailona SMA, ʻo ka mea maikaʻi loa ma mua o ka hiki ʻana o kāu keiki i 2 mau makahiki. ʻOi aku ka maikaʻi o ka mālama mua ʻana no ka mea hiki ke pale i ka pōʻino o ka neuron kaʻa ma mua o ka hoʻāʻo ʻana e hoʻohuli iā ia.

Inā ua ʻike ʻia kāu keiki me SMA ma o ka nānā ʻana i nā keiki hānau hou a i ʻole ka hoʻāʻo ʻana i ka genetic, he mea nui e kūkākūkā koke i nā koho lapaʻau. ʻOiai inā ʻaʻole e hōʻike ana kāu keiki i nā hōʻailona, hiki i ka hana mua ke kōkua i ka pale ʻana a i ʻole ka lohi ʻana i ka holomua o ke kūlana.

E kōkua kāu hui mālama olakino iā ʻoe e kaupaona i nā pōmaikaʻi a me nā pilikia o ka manawa lapaʻau e pili ana i ke kūlana kikoʻī o kāu keiki, me kā lākou ʻano SMA, nā hōʻailona o kēia manawa, a me ke kūlana olakino holoʻokoʻa.

Hiki i kaʻu keiki ke loaʻa nā lāʻau lapaʻau SMA ʻē aʻe ma hope o ka gene therapy?

ʻAe, hiki i nā keiki e loaʻa ana i ka onasemnogene abeparvovec ke pōmaikaʻi mai nā lāʻau lapaʻau a me nā therapies kākoʻo ʻē aʻe. Hoʻoponopono ka gene therapy i ke kumu genetic kumu o SMA, akā hiki i nā lāʻau lapaʻau hou ke kōkua i ka hoʻomaikaʻi ʻana i ka ulu ʻana o kāu keiki a me ka maikaʻi o ke ola.

Hiki nō ke kōkua i ka physical therapy, occupational therapy, a me ke kākoʻo hanu ma hope o ka gene therapy. Hiki i kēia mau lāʻau lapaʻau ke kōkua i kāu keiki e hiki i ko lākou hiki piha a mālama i nā pōmaikaʻi i loaʻa mai ka gene therapy.

E hoʻomohala kāu hui mālama olakino i kahi hoʻolālā mālama piha e hiki ke komo i nā ʻano hana he nui e kākoʻo i ka ulu ʻana a me ka maikaʻi o kāu keiki. ʻO ka pahuhopu ka hāʻawi ʻana i kāu keiki i nā hopena maikaʻi loa ma o ka hui pū ʻana o nā lāʻau lapaʻau i hoʻopilikino ʻia i kā lākou pono.