Nyu op fɔ Duchenne Muscular Dystrophy (DMD): Lɛ wi lan bɔt di mɛrɛsin we dɛn kɔl Duvyzat?

a andastan aw i mohs hevi en frayd we yu dokta tehl yu se yu pikin geht Duchenne muscular dystrophy (DMD). Dis na sik we pɔsin kin gɛt we i kam pan bɔy pikin dɛn mɔ ɛn smɔl smɔl i kin mek dɛn mɔsul dɛn wik. Bɔrku tɛm, di sayn dɛm kin sho wae dɛn smɔl. No kɔmplit mɛrɛsin nɔ de fɔ DMD yet. Bɔt nɔ wɔri. Mɛdikal sayɛns de go bifo ɛvride. Wit da advansment de, wan nyu op na dis nyu drug we dem kol Duvyzat. Lɛ wi tɔk bɔt am simpul wan.

Wetin na Duvyzat? Aw i kin wok?

Fɔ tɔk am simpul wan, wi mɔsul dɛn nid fɔ de ɔltɛm fɔ mek dɛn kɔntinyu fɔ strɔng. Tink bɔt di mɔsul dɛn na wi bɔdi lɛk se dɛn tan lɛk brik wɔl. Fɔ mek dis wɔl strɔng ɛn fɔ tay togɛda, i nid fɔ gɛt gud layt we dɛn kɔl mɔta. Semweso, fɔ mek wi mɔsul sɛl dɛn strɔng, wan spɛshal prɔtin we dɛn kɔl distrofin , we tan lɛk mɔta, impɔtant.

Duchenne muscular dystrophy (DMD) na wan jεnεtik kכndyushכn we de apin we di bכdi nכ de prodyuz inof pan di dystrophin protin, nכ de prodyuz inof pan am, כ nכ de prodyuz inof pan am. we dis ‘glu’ dכn lכs, di mכsul wכl de wik, i de brok dכn εn dεn nכ kin ripεr am. Na dat mek di mɔsul dɛn kin wik as tɛm de go.

apat frכm dis prכblεm, pikin dεm we gεt DMD gεt כva aktiv εnzym dεm we dεn kכl histone deacetylases (HDACs) . Dis kin mek di mɔsul dɛn gɛt inflamɛns we nɔ de dɔn . dis kin mek di mכsul dεm rεpεr mכr, εn fεt εn ska tisu kin tek di mכsul in ples.

Te naw, dɔktɔ dɛn dɔn de yuz stɛroyd, fizik tritmɛnt, ɛn tin dɛn we de ɛp pipul dɛn lɛk wilchia fɔ trit DMD. Bɔt Duvyzat na di fɔs drɔg we nɔ gɛt stɛroyd. i de wok bay we i de kכntro di כva aktiv HDAC εnzym. Dis kin mek di mɔsul dɛn nɔ inflamɛns ɛn i kin mek dɛn brok. Dis min se di spid we di mɔsul dɛn wik kin slo.

Aw a fɔ yuz dis mɛrɛsin?

Duvyzat na likwid mɛrɛsin we yu kin tek wit yu mɔt. Dɛn fɔ tek am tu tɛm insay di de, na mɔnin ɛn nɛt, wit it .

• I rili impɔtant fɔ tek di mɛrɛsin di sem tɛm ɛvride .
• Bifo yu yuz di mɛrɛsin bɔtul, yu fɔ shek am fayn fɔ at le 30 sɛkɔn .
• Mekɔp di mɛrɛsin, yuz di spɛshal sirinj we de kam wit am.
• Di tin we impɔtant pas ɔl: Nɔ miks dis mɛrɛsin wit wata ɔ ɛni ɔda wata.

Yu dɔktɔ kin gi yu dis mɛrɛsin in wan, ɔ i kin gi am wit stɛroyd. Dɛn go disayd am bay di pikin in kɔndishɔn.

Aw dɛn dɔn stɔdi bɔt di prɔpati dɛn we dis mɛrɛsin gɛt?

Insay wan big stɔdi fɔ chɛk aw dis mɛrɛsin de wok ɛn sef, dɛn bin sheb bɔy pikin dɛn we gɛt DMD to tu grup dɛn.

1. Fɔs grup: Dɛn gi dɛn di drɔg we dɛn kɔl Duvyzat.

2. Grup 2: Dɛn gi dɛn sɔntin we tan lɛk mɛrɛsin, bɔt nɔ gɛt ɛni aktif ingridiɛnt insay. Insay mɛrɛsin, wi kin kɔl dis ‘plesɛbo’ .

Dɛn kin du dis fɔ no di tru tin we di mɛrɛsin gɛt, aw i difrɛn frɔm we i jɔs de de. Ɔl di wan dɛn we tek pat pan dis stɔdi na bin bɔy pikin dɛn. Dɛn avrej ej na bin 9 ia.

Afta dɛn gi dɛn tritmɛnt ya fɔ 72 wik, dɛn bin mɛzhɔ di chenj dɛn we di pikin dɛn ebul fɔ du. Dɛn bin yuz bɔku ɔda tɛst dɛn fɔ dis.

• North Star Ambulatory Assessment (NSAA) score: Na we fɔ mɛzhɔ di ebul we pɔsin we gɛt DMD ebul fɔ du ɛvride wok.
• Siks minit waka tɛst: Si aw fa yu kin waka insay 6 minit.
• MRI tεst: De mכsu di chenj dεm we de na di fεt dεm we de na di mכsul dεm na di tכŋ εria.

Wetin na di tin dɛn we dɛn bin du we dɛn du di stɔdi?

Di tin dɛn we kin apin kin rili ɛnkɔrej wi. DMD na wan sik wae de sik kin wɔs as tɛm de go. Wetin bin klia na dis stɔdi na dat di rit we di pikin dɛn we tek Duvyzat bin de pwɛl bin slo pas di pikin dɛn we nɔ bin tek di mɛrɛsin (di plesibo) .

• Strɔng ɛn muvmɛnt: Di tɛm we i tek fɔ klaym 4 flayt stɛp dɛn bin go ɔp bay 1.25 sɛkɔn nɔmɔ afta 72 wiks pan di pikin dɛn we tek Duvyzat. Bɔt i tek lɛk 3 sɛkɔn mɔ pan di pikin dɛn we nɔ tek di drɔg. Dat min se Duvyzat slo di dɛklin pan dɛn abiliti fɔ klaym stej bay lɛk 40%.
• NSAA skɔ: We di stɔdi dɔn, di dɛkɛshɔn pan NSAA skɔ bin rili smɔl pan pikin dɛn we tek Duvyzat pas di wan dɛn we nɔ tek di drɔg. Dis sho bak se di rεt we di mכsul dεm trεnk de dכn bin rεdכks.

Dɛn tin ya kin difrɛn fɔ yu pikin, so yu fɔ tɔk to yu dɔktɔ bɔt di bɛnifit ɛn prɔblɛm dɛn we dis mɛrɛsin gɛt bifo yu disayd fɔ du sɔntin.

Sayd ɛfɛkt ɛn aw fɔ mɛn dɛn?

Lɛk ɛni mɛrɛsin, Duvyzat kin gɛt sayd ɛfɛkt. I impɔtant fɔ no bɔt dis.

Nɔto dɛn tin ya nɔmɔ de sho se pɔsin gɛt dis sik. If yu gɛt ɛni ɔda sayn we nɔ kɔmɔn, tɔk to yu dɔktɔ bɔt am.

Prɔblɛm kin de wit ɔda mɛrɛsin dɛn?

Yes, Duvyzat kin intarakt wit sɔm ɔda mɛrɛsin dɛn. So, i impɔtant fɔ tɛl yu dɔktɔ bɔt ɔl di mɛrɛsin dɛn we yu pikin de tek, lɛk di mɛrɛsin dɛn we dɛn kin gi yu, di mɛrɛsin dɛn we dɛn kin bay na kɔt (OTC), vaytamɛn dɛn, ɛn ɛbul prɔdak dɛn.

Speshali, tɛl yu dɔktɔ if yu de tek dɛn mɛrɛsin ya:

• Sɔm mɛrɛsin dɛn we de mek pɔsin nɔ gɛt bɔku kɔlɔstrel (e.g. atorvastatin, simvastatin) .
• Dayabitis mɛrɛsin lɛk mɛtfɔmin
• Mεdikeshכn dεm we de afekt di at ritm (e.g. amiodarone) .
• Sɔm antibayɔtiks (e.g. azithromycin, ciprofloxacin) .
• Sɔm mɛrɛsin fɔ mental sik
• Sɔm mɛrɛsin fɔ alɛji

Dis list nɔ kɔmplit, so i bɛtɛ fɔ tɛl yu dɔktɔ bɔt ɔl wetin yu pikin de tek.