Duchenne Muscular Dystrophy (DMD) tana beiseina thar: Duvyzat damdawi chungchang hi i zir dawn em ni?

I doctor-in i fain Duchenne muscular dystrophy (DMD) a vei tih a hrilh che chuan a rit leh hlauhawm zia chu ka hrethiam. Hei hi genetic disease a ni a, mipa naupang a nghawng ber a, zawi zawiin an taksa ruh a tichak lo. Naupan lai atanga a lan chhuah dan tlangpui. DMD hi damdawi kimchang a la awm lo. Mahse, lungngai suh. Medical science hi ni tin hma a sawn zel a. Chu hmasawnna nen chuan beiseina thar chu he damdawi thar Duvyzat tih hi a ni. A awlsam zawngin sawi ila.

Duvyzat chu eng nge ni? Engtin nge a thawh?

A awlsam zawngin, kan taksa ruhte hi a chak reng theih nan enkawl reng a ngai a ni. Kan taksa ruh (muscles) te hi brick wall ang maiin han ngaihtuah teh. He bang hi a nghet leh inzawm tlat tur chuan mortar layer tha tak a mamawh a ni. Chutiang bawkin, kan taksa ruh cell-te chak taka awm reng tûr chuan, protein bik dystrophin , mortar ang maia awm chu a pawimawh hle.

Duchenne muscular dystrophy (DMD) hi taksain dystrophin protein a siam chhuah tlem emaw, a siam chhuah tlem emaw, a siam chhuah tlem emaw a nih chuan genetic condition a ni. He ‘glue’ hi a bo chuan taksa ruh (muscle wall) a chak lo a, a chhe a, siamthat theih a ni lo. Chuvang chuan hun kal zelah taksa ruh (muscles) a chak lo thin.

He harsatna bakah hian DMD vei naupangte hian histone deacetylases (HDACs) tih enzymes overactive an nei bawk. Hei hian taksa peng hrang hrangah chronic inflammation a thlen thin. Hei hian taksa ruh siamthatna a ti tlem lehzual a, taksa ruh chu thau leh scar tissue-in a thlak ta a ni.

Tun thleng hian doctor-te chuan DMD enkawlna atan hian steroid, physical therapy, leh wheelchair ang chi assistive devices te an hmang tawh a ni. Mahse Duvyzat hi nonsteroidal damdawi hmasa ber a ni. Overactive HDAC enzyme control-in hna a thawk a ni. Hei hian taksa peng hrang hranga inflammation a tihziaawm a, a chhe thei bawk. Chumi awmzia chu taksa chak lohna speed chu a slow tihna a ni.

He damdawi hi engtin nge ka hman ang?

Duvyzat hi damdawi tui tak a ni a, i ei tur a ni. Ni khatah vawi hnih, zing leh zanah ei tur a ni a, ei tur nen .

• Nitin damdawi hi a ruala ei a pawimawh hle .
• Damdawi bottle i hman hmain second 30 tal i vawt tha tur a ni .
• Damdawi chu teh la, a kaihhnawih syringe bik hmangin teh rawh.
• A pawimawh ber: He damdawi hi tui emaw, tui dang emaw nen chawhpawlh suh.

I doctor hian he damdawi hi a pe mai thei a, steroid nen pawh a pe thei bawk. Naupang dinhmun a zirin thutlukna siam a ni ang.

Engtin nge he damdawi property hi zirchian a nih?

He damdawi hman tangkai dan leh a him leh him loh enfiahna zirchianna lian takah chuan DMD vei mipa naupangte chu group hnih ah an inthen a.

1. Pawl khatna: Damdawi Duvyzat an pe a.

2. Group 2: Damdawi ang deuh, mahse a chhunga active ingredient nei lo thil pek an ni. Damdawi lamah chuan hei hi ‘placebo’ kan ti thin.

Hei hi damdawiin a nghawng dik tak, a awm mai mai nen a danglamna tehna atan tih a ni. He zirchiannaa tel zawng zawng hi mipa naupang an ni. An kum zat chu kum 9 a ni.

Heng enkawlnate hi kar 72 chhung an pek hnuah naupangte theihna inthlak danglamna chu an teh a ni. Hemi atan hian test dang eng emaw zat hman a ni bawk.

• North Star Ambulatory Assessment (NSAA) score: DMD vei mi pakhatin nitin hna a thawh theih dan tehna hmanrua a ni.
• Minute ruk chhunga kal test: Minute 6 chhunga i kal theih dan tur a en thin.
• MRI test: Thigh area muscle-a thau awm zat inthlak danglamna a teh thin.

He zirchianna atanga chhuak chu eng nge ni?

A rah chhuah chu a fuh tak zet a ni. DMD hi hun kal zelah symptoms a zual zel thin a ni. He zirchiannaa chiang tak chu Duvyzat ei naupangte zingah hian damdawi (the placebo) ei lo naupangte aiin a tlahniam zawk tih hi a ni.

• Chakna leh kal theihna: Duvyzat ei naupangte zingah chuan stairs flight pali chhoh hun chhung chu kar 72 hnuah second 1.25 chauhin a pung a ni. Mahse, damdawi ei lo naupangteah chuan second 3 vel a la rei zawk. Chumi awmzia chu Duvyzat hian an stairs chhoh theihna tlahniam chu 40% velin a ti hniam tihna a ni.
• NSAA score: Zirchianna tawp lamah chuan Duvyzat ei naupangte zingah NSAA score tlahniam chu damdawi ei lote aiin a tlem zawk tih a chiang hle. Hei hian taksa chakna tlahniam rate a tlahniam tih a tilang bawk.

Heng result te hi i fa tan a inang lo thei a, chuvangin thutlukna i siam hmain he damdawi hi a thatna leh a hlauhawmna te chu i doctor nen in sawiho hmasa phawt tur a ni.

Side effects leh engtin nge kan enkawl ang?

Damdawi dang ang bawkin Duvyzat hian side effect a nei thei a ni. Hei hi hriat chian a pawimawh hle.

Hengte chauh hi a symptoms a ni lo. Symptom danglam dang i neih chuan i doctor nen sawiho rawh.

Damdawi dangah harsatna a awm thei em?

Ni e, Duvyzat hian damdawi dang thenkhat nen a inzawm thei. Chuvang chuan i fain damdawi a ei zawng zawng, prescription medication, over-the-counter (OTC) damdawi, vitamin, leh herbal products te chu doctor hnenah hrilh vek a pawimawh.

A bik takin, a hnuaia damdawi te hi i ei a nih chuan i doctor hnenah hriattir tur a ni:

• Cholesterol tihhniamna damdawi thenkhat (e.g. atorvastatin, simvastatin) .
• Zunthlum damdawi metformin ang chi te
• Heart rhythm tichhe thei damdawi (e.g. amiodarone) .
• Antibiotics thenkhat (e.g. azithromycin, ciprofloxacin) te hi a tha hle.
• Rilru natna damdawi thenkhat
• Allergy natna damdawi thenkhat

He list hi a kim lo a, chuvangin i fain a ei zawng zawng chu doctor hnenah hrilh vek a tha ber.