He aha te Risdiplam: Ngā Whakamahinga, te Inenga, ngā Pānga taha me ētahi atu

Ko te Risdiplam he rongoā rāpoi e āwhina ana ki te rongoa i te atrophy uaua tuaiwi (SMA), he āhua ira onge e pā ana ki te kaha o te uaua me te nekehanga. E mahi ana tēnei rongoā mā te āwhina i tō tinana ki te whakaputa i te pūmua e hiahia ana ngā neuron motuka kia pai te mahi, ka taea e tēnei te whakaiti i te ngoikore o te uaua, ā, ka āwhina pea ki te whakapai ake i ētahi mahi uaua i te roanga o te wā.

He aha te Risdiplam?

Ko te Risdiplam he rongoā waha i hangaia hei rongoa i te atrophy uaua tuaiwi i roto i ngā tamariki me ngā pakeke. No te kāwai rongoā e kīia nei ko ngā whakarerekē i te SMN2 splicing, he uaua tēnei engari ko te tikanga he āwhina i tō tinana ki te whakamahi pai i ngā tohutohu ira e mau ana ki te hanga neuron motuka hauora.

Ka tae mai tēnei rongoā hei otinga wai ka tangohia e koe mā te waha, e māmā ake ai te hoatu ki ngā pēpi me ngā tamariki nohinohi e uaua ana ki te horo i ngā pire. Ka taea hoki e te puka wai te inenga tika i runga i te taumaha o tō tinana, he mea nui tēnei mō ngā tamariki e tipu ana.

He tohu nui te Risdiplam i roto i te rongoā SMA nā te mea ko te rongoā waha tuatahi e wātea ana mō tēnei āhua. Kāore i rite ki ētahi atu rongoā SMA e hiahia ana ki te werohanga ki roto i te tuaiwi, ka taea e koe te tango i te risdiplam i te kāinga me tō rāpoi mahi.

He aha te Risdiplam e whakamahia ana?

Ka whakamahia te Risdiplam hei rongoa i te atrophy uaua tuaiwi i roto i ngā tūroro e 2 marama te pakeke, neke atu rānei. Ko te SMA he āhua ira e kore ai ngā neuron motuka (ngā pūtau io e whakahaere ana i te nekehanga o te uaua) e mahi tika ana, e arahi ana ki te ngoikore o te uaua me te moumou i te roanga o te wā.

Kua whakaaetia te rongoa mo nga momo SMA katoa, mai i nga momo tino kaha e pa ana ki nga kohungahunga ki nga momo ngawari kaore pea e whakaatu tohu tae noa ki muri i te tamarikitanga, i te pakeketanga ranei. Ko tenei whakaaetanga whanui te tikanga ahakoa ahea i puta tuatahi ai nga tohu SMA, te kaha ranei, tera pea he whiringa maimoatanga a risdiplam e tika ana kia korerohia me to taakuta.

Ka tohutohu pea to taakuta i te risdiplam mena kua kitea koe, to tamaiti ranei he SMA na roto i nga whakamatautau ira. Ko te wawe ka timata te maimoatanga, ka pai ake nga hua ka puta, koinei te take e timata ai nga taakuta i tenei rongoa i te wa e taea ai i muri i te tātaritanga.

Me pehea te mahi a Risdiplam?

Ka mahi a Risdiplam ma te whai i te take o te SMA i te taumata ira. Ko nga tangata e mate ana i te SMA he raruraru ki te ira e kiia nei ko SMN1, engari he ira taapiri ano to ratou e kiia nei ko SMN2 ka taea te mahi kotahi, engari kaore i te pai.

Whakaarohia me te mea he wheketere matua kua pakaru, engari he wheketere taapiri iti ake ka awhina mena ka kaha ake te mahi. Ko te tikanga, ka awhina a Risdiplam i taua wheketere taapiri (te ira SMN2) kia pai ake te mahi, kia nui ake te pūmua e hiahia ana nga neuron motuka kia noho ora.

Ko tenei rongoa e kiia ana he ngawari te whai hua, kaua ki te rongoa, ko te tikanga ka taea e ia te whakaroa i te ahunga whakamua o te ngoikoretanga o te uaua, a tera pea ka pai ake etahi mahi, engari kaore e tino huri i te ahua. Ka piki haere nga paanga i roto i te waa, no reira kaore pea koe e kite i nga huringa tonu.

Ko te nuinga o nga tangata e pai ana ki te risdiplam ka kite i nga whakapainga i roto i to raatau kaha ki te horomia, ki te manawa, me te neke i roto i nga marama tuatahi e 6 ki te 12 o te maimoatanga. Heoi, he rereke te urupare a te katoa, a ka aroturukihia e to taakuta to ahunga whakamua i nga wa katoa kia kite i te pai o te mahi o te rongoa mo koe.

Me pehea taku tango i te Risdiplam?

Ka tae mai a Risdiplam hei otinga wai e inu ana koe i te waha kotahi i te ra, ā, he mea nui kia inu i te wā kotahi i ia rā kia mau tonu ai ngā taumata i tō tinana. Ka taea e koe te inu me te kore kai rānei, engari mā te inu me te kai ka āwhina i te whakaiti i te pōhara o te puku.

Me inu te rongoā mā te whakamahi i te syringe waha e tae mai ana me tō rongoā, nā te mea ka whakarite tēnei kia whiwhi koe i te horopeta tika i whakaritea e tō tākuta. Ki te hoatu ana e koe ki te tamaiti, ka taea e koe te whakaranu i te horopeta ki te wai iti, te miraka, te tātai rānei, engari me mōhio kei te inu rātou i te ranunga katoa tonu.

Anei ngā mea e hiahia ana koe ki te mōhio mō te inu i te risdiplam i te haumaru:

• Whakaorangia te rongoā i roto i te pouaka whakamātao, ā, whakamahia i roto i ngā rā 64 mai i te whakatuwheratanga tuatahi
• Whakaoho marie i te pounamu i mua i ia horopeta kia mōhio kei te pai te whakaranu o te rongoā
• Inumia te horopeta i te pōturi, ina koa kei te raru koe ki te horomia
• Kaua e whakaranu i te horopeta ki ngā kai waikawa, ki ngā inu rānei pēnei i te wai karaka, nā te mea ka pā tēnei ki te mahi o te rongoā
• Ki te whakamahi ana koe i te ngongo whāngai, pātai atu ki tō rōpū hauora mō ngā tohutohu motuhake

Ka tātai tō tākuta i tō horopeta tika i runga i tō taumaha tinana, ā, me whakatika tēnei horopeta i te wā e tipu ana koe, tō tamaiti rānei. Kaua rawa e huri i te horopeta i runga i a koe anō, ahakoa ki te whakaaro koe kāore i te pai te mahi.

Kia Pēhea Te Roa E Inu Ai Au i te Risdiplam?

Ko te Risdiplam te tikanga he rongoā wā roa e hiahia ana koe ki te inu tonu i te wā e āwhina ana, ā, e pai ana koe ki te inu. I te mea he āhuatanga mau tonu te SMA, ko te whakamutu i te rongoā ka āhei te ngoikore o te uaua ki te piki anō.

E tohutohu ana te nuinga o ngā tākuta kia haere tonu te risdiplam mō te wā mutunga kore, i te wā e kite ana koe i ngā painga, ā, kāore koe e pā ana ki ngā pānga taha nui. E kite ana ētahi tāngata i ngā whakapainga i roto i ngā marama tuatahi, i te wā e roa ana pea te tau ki ētahi atu ki te kite i ngā huringa nui i roto i te mahi uaua, i te oranga whānui rānei.

Ka arotakehia e tō rōpū hauora i ngā wā katoa te pai o te mahi a te rongoā mā roto i ngā momo whakamātautau me ngā aromatawai. Ko ēnei pea ko ngā whakamātautau mahi uaua, ngā aromatawai manawa, me ngā ine kounga o te oranga hei whakatau mēnā ko te risdiplam tonu te rongoā tika māu.

Mēnā kei te whakaaro koe ki te whakamutu i te risdiplam, he mea nui kia kōrerohia tēnei ki tō tākuta i te tuatahi. Ka taea e rātou te āwhina i a koe ki te whakatau i ngā painga kua riro i a koe ki ngā pānga taha pea e pā ana ki a koe, me te tūhura i ngā kōwhiringa rongoā kē atu mēnā e tika ana.

He aha ngā pānga taha o te Risdiplam?

Pērā i ngā rongoā katoa, ka taea e te risdiplam te pānga taha, ahakoa kāore te katoa e pā ana ki a rātou. Ko ngā pānga taha tino noa he ngawari noa iho, ā, ka pai ake i te wā e whakatika ana tō tinana ki te rongoā i ngā wiki tuatahi o te rongoā.

Ko ēnei ngā pānga taha tino pōti e pā ana ki a koe:

• Kirika, e puta ana i te 20% pea o te hunga e tango ana i te risdiplam
• Korere, he tūru rānei
• Pānga kiri, he riri kiri rānei
• Ngā mate o te ara rārangi o runga pērā i te makariri
• Ngā pōrearea, he mamae puku rānei
• Ānini
• Ngenge, he ngenge rānei atu i te tikanga

Ko ēnei pānga taha noa e taea ana te whakahaere, ā, kāore e hiahiatia te whakamutu i te rongoā. Ka taea e tō tākuta te whakaaro i ngā huarahi hei whakaiti i te pōrearea, pērā i te tango i te rongoā me te kai mō te pōrearea puku, te whakamahi rānei i ngā rongoā whakaheke kirika i te wā e tika ana.

Ko ngā pānga taha nui ake he iti noa iho, engari e hiahiatia ana te āwhina hauora tonu. Whakapā atu ki tō tākuta i te wā tonu mēnā ka pā ana ki a koe ngā pānga mate pāwera nui, te kirika nui tonu, te korere nui e arahi ana ki te whakawātea wai, he huringa rānei e pā ana ki te manawa, te horomia rānei.

He āwangawanga mō ngā pānga wā-roa

Kei te āwangawanga ētahi tāngata mō ngā pānga wā-roa, nā te mea he rongoā hou a risdiplam. Ahakoa kei te kohia tonutia ngā raraunga wā-roa, e whakaatu ana ngā rangahau o nāianei he haumaru tonu te rongoā, he whai hua hoki mō te nuinga o ngā tāngata i roto i ngā tau maha o te maimoatanga.

Ko wai kāore e tika kia tango risdiplam?

Kāore a Risdiplam e tika ana mō te katoa, ā, ka āta aromatawai tō tākuta mēnā he haumaru mō koe i runga i tō hauora whānui me tō hītori rongoā. Kāore te rongoā i te tūtohutia mō ngā tāngata kua pā ki a rātou ngā pānga mate pāwera nui ki a risdiplam, ki ētahi atu rānei o ōna whakauru.

Ko ngā tāngata e pā ana ki ētahi āhuatanga hauora, me nui ake te aroturuki, tērā pea kāore rātou e tika ana mō te rongoā risdiplam. Ka tino tūpato tō tākuta mēnā he raruraru nui tō pūkahukahu, tō ate rānei, nā te mea ka āwhina ēnei okana ki te tukatuka i te rongoā.

He mea nui kia whakaarohia mō ētahi rōpū tāngata:

• Me kōrero ngā wāhine hapū, ngā wāhine whāngai ū rānei i ngā tūponotanga me ngā painga ki tō rātou tākuta
• Ngā tāngata he hītori o ngā pānga mate pāwera nui ki ngā rongoā
• Ko te hunga e tango ana i ētahi atu rongoā ka pā tahi pea ki a risdiplam
• Ngā tāngata he uaua nui ki te horomia, tērā pea he uaua ki a rātou te tango i ngā rongoā waha i runga i te haumaru
• Ngā tāngata e pā ana ki ngā mate kaha, nui, tērā pea ka kino ake i te rongoā

Mēnā he āwangawanga ōna mō te tika o risdiplam mō koe, kaua e whakaroa ki te kōrero tuwhera ki tō rōpū hauora. Ka āwhina rātou i a koe ki te mārama ki ngā painga me ngā tūponotanga i runga i tō āhuatanga motuhake.

Ingoa waitohu o Risdiplam

E hokona ana a Risdiplam i raro i te ingoa waitohu Evrysdi, e hangaia ana e Genentech, he pūtea o Roche. Koinei anake te ingoa waitohu e wātea ana a risdiplam i tēnei wā, nā te mea kāore anō ngā putanga whānui i te mākete.

I whakaaetia a Evrysdi e te FDA i te tau 2020, he rongoa hou tonu i roto i te ao rongoa mo te SMA. He rite tonu te ingoa waitohu puta noa i te ao, ahakoa ka rereke pea te waatea i ia whenua i runga i nga whakaaetanga ture o te rohe.

I te wa e korero ana koe i tenei rongoa ki to roopu hauora, ki te kamupene inihua ranei, ka taea e koe te korero ki a ia ko risdiplam, ko Evrysdi ranei. E rua nga ingoa e tohu ana i te rongoa kotahi, engari ko nga puka inihua me nga whakahaunga ka whakamahi i te ingoa waitohu Evrysdi.

Nga Kōwhiringa kē mō Risdiplam

Ahakoa he whiringa maimoatanga nui a risdiplam mo te SMA, ehara koinei anake te mea e wātea ana. Ka whakaaro pea tō tākuta ki ētahi atu maimoatanga i runga i tō momo SMA, tō tau, tō hauora whānui, me ō hiahia ake.

Ko nga whiringa kē nui ki a risdiplam ko te nusinersen (Spinraza), e tukuna ana hei werohanga ki roto i te wai roro i ia marama, me te onasemnogene abeparvovec (Zolgensma), he rongoa ira kotahi-wa e tukuna ana mā te uaua. He rerekē te mahi a ia maimoatanga, ā, he pai me ōna ake whakaaro.

Anei te whakataurite o enei whiringa kē ki a risdiplam:

• Me werohia a Nusinersen ki te tuara i ngā wā katoa, engari kua roa kē e wātea ana, ā, he nui ake ngā raraunga mō te wā roa
• He maimoatanga kotahi-wa te rongoa ira, engari kua whakaaetia anake mō ngā tamariki tino pōtiki me ētahi momo SMA
• Ka whai wātea a Risdiplam i te waha, engari ka puhoi ake te mahi i ētahi atu whiringa
• He mea nui tonu te manaaki tautoko tae atu ki te rongoa tinana, te tautoko manawa, me te whakahaere kai ahakoa te rongoa e whiriwhiria ana e koe

Ka āwhina tō tākuta i a koe ki te mārama ko tēhea huarahi maimoatanga ka pai mō tō āhuatanga. Ka whakamahi pea ētahi tāngata i ngā huinga maimoatanga, ka huri rānei mai i tētahi ki tētahi i te roanga o te wā i runga i tō rātou urupare me ngā hiahia e huri ana.

He Pai Ake A Risdiplam I Te Nusinersen?

He rongoā whai hua a Risdiplam me te nusinersen mō te SMA, engari he rerekē te āhua o tā rāua mahi, ā, he rerekē anō ngā painga. Kāore tētahi e pai ake ana i tētahi, te nuinga o te wā ka whakawhirinaki te whiriwhiri ki ō āhuatanga me ō hiahia.

Ko te painga nui o Risdiplam he mea tango waha i te kāinga, he mea māmā ake i te nusinersen, e hiahia ana kia haere tonu ki te whare hauora mō ngā werohanga tuarā. He mea nui tēnei mō ngā tāngata e uaua ana ki te haere, e hiahia ana rānei ki te whakaiti i ngā mahi rongoā.

Ko te nusinersen, i tērā taha, kua roa kē e wātea ana, ā, he nui ake ngā raraunga mō te wā roa e whakaatu ana i ōna hua. E ai ki ētahi rangahau, tērā pea ka tere ake te mahi i te risdiplam ki te whakapai ake i ētahi mahi uaua, inā koa ki ngā tāngata e kaha ana te SMA.

Ko te whakatau i waenga i ēnei rongoā ka heke iho ki ngā whakaaro whaihua pēnei i tō whakamarie ki ngā werohanga, tō uru ki ngā whare hauora motuhake, me te pai o tō urupare ki ngā rongoā o mua. Ka tīmata ētahi tāngata ki tētahi rongoā, ā, muri iho ka huri ki tētahi atu i runga i ō rātou wheako me ngā hiahia e huri ana.

Ngā Pātai Auau mō Risdiplam

He Haumaru a Risdiplam mō ngā Tāngata e Pā ana ki ngā Pānga o te Ngākau?

Ka taea te whakamahi i te Risdiplam i te nuinga o te wā i roto i ngā tāngata e pā ana ki ngā pānga o te ngākau, engari ka hiahia tō tākuta ki te aroturuki i a koe. Ka pā te SMA ki ngā uaua e whai wāhi ana ki te hā me te tohanga toto, nō reira me mahi tahi tō tākuta ngākau me tō tākuta roro kia mōhio ai he tika te rongoā mōu.

Ka whanake ētahi tāngata e pā ana ki te SMA i ngā raruraru ngākau i te roanga o te wā, engari e whakaatu ana ngā tohu o nāianei kāore te risdiplam e kino ake i ngā raruraru ngākau, ā, tērā pea ka āwhina mā te whakapai ake i te mahi uaua whānui. Tērā pea ka hiahia tō tākuta ki te mahi i ngā whakamātautau mahi ngākau i ngā wā katoa kia mōhio ai kei te pūmau ngā mea katoa.

Me aha au mēnā ka tango au i te nui rawa o te Risdiplam?

Mēnā i tūpono koe ka nui ake te risdiplam i te mea i tohua, whakapā atu ki tō tākuta, ki te pokapū whakahaere paitini rānei i te wā tonu, ahakoa kāore koe e pōuri ana i te wā tonu. Ko te nui rawa o te tango ka piki ake pea tō tūponotanga ki ngā pānga taha, ahakoa he onge ngā horopeta nui ki tēnei rongoā.

Kaua e ngana ki te "whakatika" i te horopeta nui mā te karo i tō horopeta e whai ake nei, mā te tango rānei i te iti. Engari, whai i ngā tohutohu a tō tākuta mō te wā hei tīmatanga anō i tō hōtaka horopeta noa. Puritia te rongoā ki tētahi wāhi haumaru e kore ai e tūpono ngā horopeta nui.

Me Pēhea Taku Mahi Mēnā Kua Ngaro i Ahau tētahi Horopeta o Risdiplam?

Mēnā kua ngaro i a koe tētahi horopeta o risdiplam, tangohia i te wā tonu ka mahara koe, ki te kore e tata ana te wā mō tō horopeta e whai ake nei. I tērā wā, karohia te horopeta i ngaro, ka haere tonu ki tō hōtaka auau. Kaua rawa e tango e rua ngā horopeta i te wā kotahi hei whakatika i tētahi horopeta i ngaro.

Kāore he tūponotanga mēnā ka ngaro i a koe tētahi horopeta, engari ngana ki te pupuri i te horopeta o ia rā mō ngā hua pai. Mēnā he maha ngā wā ka wareware koe i ngā horopeta, whakaarohia te whakatakoto i ngā whakamaharatanga waea, te whakamahi rānei i tētahi kaiwhakarite pire hei āwhina i a koe ki te mahara.

Āhea Āhau e Kati ai te Tango i te Risdiplam?

Me kati anake e koe te tango i te risdiplam i raro i te ārahitanga a tō tākuta, nā te mea ko te kati ohorere ka taea e ō tohumate SMA te kino anō. Ko te nuinga o te tangata me haere tonu te tango i tēnei rongoā mō te wā roa kia mau tonu ai ngā painga i riro mai i a rātou.

Ka tūtohu pea tō tākuta kia kati koe i te risdiplam mēnā ka whanake koe i ngā pānga taha nui e kore e pai ake, mēnā ka mutu te mahi whai hua o te rongoā, mēnā ka wātea mai tētahi kōwhiringa maimoatanga pai ake rānei. Mā rātou koe e āwhina kia huri haumaru ki ngā maimoatanga kē mēnā e tika ana.

Ka Taea e Āhau te Haere me te Risdiplam?

Āe, ka taea e koe te haere me te risdiplam, engari me whakamahere marie koe kia tika te penapena i te rongoa me te whakarite kia kore koe e pau i te wa e ngaro ana koe i te kainga. I te mea me whakamatao te risdiplam, ka hiahia koe ki te whakamatao kawe mo te haere me te uru ki te whakamatao i to haerenga.

Me kawe mai he rongoa taapiri hei take mo nga whakaroa haerenga, me te kawe mai i tetahi reta mai i to taakuta e whakamarama ana i to hiahia mo te rongoa. Mena kei te rere koe, waiho te rongoa ki roto i to peeke kawe kia kore ai e ngaro, e pa ana ranei ki nga wera nui i roto i nga tueke kua tirohia.