A châng chuan kan gene-a inthlâk danglamna tenau te hian hriselna lama harsatna lian tak a thlen thei a ni. hATTR amyloidosis hi chutiang thil awm lo tak, mahse genetic condition na tak tak zinga mi a ni. Hetah hian kan thin atanga protein siam chu dik lo takin a pungkhawm a, nerve te a tichhe thin. Hei hian harsatna tam tak a thlen thei a, chu chu kal harsa, natna, leh rilru hahna te hi a ni. Mahse tunah chuan hei hi enkawl dan thar a awm ta. Vawiin hian he damdawi thar Wainua tih hi han sawi ila.
Damdawi Wainua hian engtin nge hna a thawh?
Hei hi hrethiam tur chuan hATTR natna hi awlsam takin han thlir hmasa ila. Kan taksa hian protein pakhat transthyretin (TTR) a nei a. Hei hi kan thin (liver) siam a ni. hATTR natna vei zinga TTR gene inthlak danglamna tlemte avang hian he protein hi a fold dik lo a, a inzawm khawm a, taksaah ‘amyloid’ an tih clump angin a deposit a ni. Curdling milk ang mai a ni. Heng amyloid clump te hi nervous system ah an awm tam ber. Tichuan nerve te a chhiat a, an hnathawh a tichhe thin.
Tunah chuan Wainua vaccine hian eng nge a tih tih hi han en ila. Damdawi Wainua hian TTR protein dik lo siamtu gene chu direct-in a target a ni. A awlsam zawngin, thupek dik lo pe tu gene chu a ‘silence’ a ni.
Hei hian liver atanga TTR protein pangngai lo siam chhuah zat a tihtlem phah a ni. Hei hian a lehlamah nerve-a amyloid awm zat a tihtlem a, nerve chhiatna kal zel dan tur control-na kawngah a pui bawk.
Hei hi thla tin injection a ni a, vun hnuaiah inject thin a ni. Thil danglam bik chu damlo emaw an enkawltu emaw in lama hman tur atana siam ‘auto-injector’-ah a awm a ni.
Engtin nge he enkawlna hi ka tan a tha em tih ka hriat theih ang?
I doctor chuan Wainua hi i tan a tha em tih a rel ang. He enkawlna hi a tlangpuiin a hnuaia mite tan hian a tha ber a ni:
- h Puitling ATTR Amyloidosis i vei tih finfiah tawh i nih chuan .
- Natna hian nerve a tichhia a nih chuan .
I doctor chuan he thutlukna a siam hmain thil engemaw zat a ngaihtuah ang.
- Chhungkaw chanchin : I chhungkuaah hian he natna hi an vei tawh em tih enfiah thin ang che.
- Symptom Check: I symptoms te uluk taka enfiah a ni ang.
- Genetic testing: TTR gene mutation finfiah nan thisen test an nei thin.
- Imaging test: MRI scan hmanga enfiah a niHetiang test hian nerve leh taksa peng dangah amyloid a awm leh awm loh a enfiah thei bawk.
Engtin nge Wainua damdawi hlawhtlinna hi enchhin a nih?
Damdawi thar eng pawh market-a tihchhuah a nih hmain, a quality, a him leh that loh hriat tumin zirchianna nasa tak neih a ni. Wainua damdawi hi NEURO-TTTransform tih hming puin clinical trial an nei tawh bawk.
He zirchianna hian a bik takin:
1. Engtin nge Wainua damdawi hian thisenah TTR protein level a tihdanglam?
2. Hei hian hATTR vei te nerve chhiatna nena inzawm symptoms te chu engtin nge a nghawng ang?
Chungte chu tehna atan hian hmanraw bik an hmang a.
- Neuropathy Impairment Score (NIS): Hei hi scoring system bik a ni. Muscle chakna, reflexes leh sensation te a teh thin. Score a hniam poh leh nerve dinhmun a tha zawk.
- Quality of Life (QOL-DN): Hei hian damlo hahna te, nitin hna a thawh theihna te, leh rilru dinhmun te a teh a ni. Hetah pawh hian score hniam zawk hian nunphung tha zawk a tilang a ni.
Wainua hlawhtlinna hi he zirchianna result leh zirchianna danga damlo, damdawi tel lo placebo pekte result nen khaikhin a ni.
| Research-a telte thlan dan | |
|---|---|
| Inclusion Criteria a ni | Exclusion Criteria a ni |
| - Kum 18-82 inkar - HATTR avanga nerve tihchhiat stage 1 (anpuina tel lova kal) emaw stage 2 (tanpuina nena kal) emaw nei te - TTR gene-a mutation awm tih finfiah tawhte | - Amyloidosis chi dang vei te - Zunthlum ang chi thil dang avanga nerve tihchhiat te - Liver transplant nei tawh emaw, nei dawn emaw te - hATTR enkawlna dang thenkhat dawng thinte |
Research atanga result lian ber berte chu engte nge ni?
He zirchianna result hian a tichak hle. Kar 66 chhung Wainua hmanga enkawlte chuan placebo dawngte aiin result a tha zawk tih a chiang hle .
- An thisenah hian TTR protein hlauhawm tak level chu a tlahniam hle tawh a ni.
- Neurological symptoms (NIS score) te chu nasa takin an control a ni.
- Tin, an nunphung pawh a lo ṭha chho tih a chiang hle bawk.
Wainua hian eng side effect nge a neih a, engtin nge kan enkawl ang?
Damdawi dang ang bawkin Wainua hian side effect tenau engemaw zat a nei thei bawk. A tlangpui ber chu:
| Side effect a awm thei | Engtin nge kan enkawl ang |
|---|---|
| Vitamin A level a tlahniam | Vitamin A supplement hi doctor-in a tih ang chiah khan ei thin ang che. I doctor-in a tih aia tam ei ngai suh. I mit hmuh danah danglamna i hmuh chuan, zan lama mit hmuhna tlahniam emaw, mit vawt emaw, doctor hnenah hrilh nghal rawh. |
| Luak chhuak | Tui leh tui tam tak in thin ang che. Chaw thau leh rit ei loh tur. Zawi zawiin ei rawh. Ni engemaw zat hnuah pawh i luak chhuak lo a nih chuan i doctor nen inbia ngei ang che. |
Hengte hi a side effect lian ber berte chu an ni. Chu bâkah, i doctor-te nên i rilru tibuaitu symptoms eng pawh chu sawipui ang che.
Engtin nge enkawlna a hlawhtlin leh hlawhtlin loh i hriat theih ang?
I doctor chuan i hriselna a enfiah fo ang. Hei hian i TTR level enfiah nan thisen test a huam ang. I neurological symptoms te chu engtiang chiahin nge a lo tha chho zel tih an enfiah bawk ang. Hun a kal zel a, i taksa ruh chakna te, nitin hna thawh theihna te, leh i rilru put hmang te a lo tha chho zel tih i hmu tan thei a ni.
A pawimawh ber chu, Doctor thurawn tel lova vaccine pek hi titawp lo la, vaccine pek hun tur pawh thlak danglam suh.
Damdawi dang emaw, ei tur dang emaw-ah harsatna a awm em?
Tuna zirchianna atanga a lan dan chuan Wainua leh damdawi dang emaw ei tur dang emaw inkarah hian inzawmna tha lo a awm lo. Mahse, nakin lawkah thu thar a lo chhuak thei a ni.
Chuvangin, he enkawlna i tan hma leh a hun lai hian i doctor hnenah damdawi dang zawng zawng, vitamin, supplement, leh Ayurvedic product i hman zawng zawngte kimchang takin hrilh rawh. Hei hi i himna atan a pawimawh hle.
Take-Home Thuchah a ni
- Wainua hi novel, thla tin vaccine hmanga enkawlna pawm a ni a, hei hi rare genetic condition hATTR Amyloidosis tan a ni.
- Hei hian taksain protein pangngai lo, nerve tichhe thei a siam chhuah a ti tlem a ni.
- Hei hian natna avanga nerve tihchhiatna control nan a pui a, damlote nunphung a tisang bawk.
- Vitamin A level tlahniam hi side effect lian tak a nih avangin doctor-in a pek angin vitamin A supplement hi ei a pawimawh hle.
- Enkawlna eng pawh i tan hma emaw, i chawlhsan hmain doctor nen inbiakna nei fo la, a nghawng tha lo a awm chuan.
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