If yu ɔ sɔmbɔdi na yu famili gɛt wan sik we dɛn kɔl Congenital Adrenal Hyperplasia (CAH), dis go bi rili impɔtant nyus fɔ yu. Afta 70 ia, dɛn dɔn gri fɔ mek wan nyu tritmɛnt we dɛn go yuz fɔ dis sik spɛshal wan naw. Dis na rili big op fɔ pipul dɛn we de liv wit CAH. So lɛ wi tek tɛm luk wetin na dis nyu mɛrɛsin, aw i de wok, ɛn wetin na di bɛnifit dɛn we i gɛt.
Fɔs, lɛ wi ɔndastand, wetin na CAH?
Fɔ tɔk am simpul wan, CAH na wan sik we pɔsin kin gɛt we i kam pan wi adrenal gland dɛn. Dɛn gland ya de ɔp wi kidni dɛn. Dɛn kin mek bɔku ɔmon dɛn we rili impɔtant fɔ wi bɔdi.
Tink bɔt di adrenal gland lɛk faktri. Di men tin we dɛn kin mek na dis faktri na wan ɔmon we dɛn kɔl kɔtisol. Dis na ɔmon we rili impɔtant we de ɛp wi bɔdi fɔ bia wit strɛs, sik, ɛn denja.
we di kכtisol lεvεl na hεlty pכsin dכn, di bren (lεk fכktri mεnaja) de sεnd wan signal (כda) we dεn kכl ACTH to di adrenal gland fכ "mek kכtisol." bכt pan pכsin we gεt CAH, di adrenal gland nכ gεt wan ki εnzym, כ ‘wokman’, we nid fכ mek kכtisol. So i nɔ mata aw bɔku ACTH di bren de sɛn, di faktri nɔ ebul fɔ mek kɔtisol.
Dis de mek wi aks tu kwɛstyɔn dɛn:
1. Di bɔdi nɔ de mek di kɔtisol we nid .
2. As di ACTH signal dεm we de kכmכt na di bren de kכntinyu fכ fכlכ, wan כda kayn כmon we di adrenal gland de mek we dεn kכl andrכjen, de kכmכt pasmak .
dis androjen dεm (e.g. tεstostεron) na man כmon dεm. Man ɛn uman ɔl tu nid sɔm pan dɛn, bɔt na CAH, dɛn kin tu ay. Dis kin mek di pikin bigin fɔ bɔn pikin kwik kwik wan, i nɔ kin ebul fɔ bɔn pikin, ɛn i kin mek i gɛt ɔda prɔblɛm dɛn wit di we aw pikin de gro .
Fɔ lɔng tɛm naw, di men tritmɛnt fɔ dis na fɔ gi stɛroyd (glucocorticoids) we de wok lɛk kɔtisol na do. dis nכ de כnli riples di kכtisol we di bכdi nכ gεt, bכt i de kכntro di ACTH signal frכm di bren, we de ridyus di prodakshכn fכ andrכjen dεm. Bɔt di prɔblɛm na dat dis kin mek pɔsin tek bɔku stɛroyd pas aw i kin tek fɔ lɔng tɛm. Dis kin mek yu gɛt bɔku bad bad tin dɛn lɛk fɔ gɛt bɔku bɔku bɔdi, fɔ mek yu bon dɛn tan, ɛn fɔ mek yu nɔ ebul fɔ slip fayn .
Aw yu bin fɛn dis nyu mɛrɛsin?
Sayɛnsman dɛn bin de wɔnda if ɔda tin de fɔ sɔlv dis prɔblɛm we gɛt fɔ du wit stɛroyd. insted fכ gi bכku stεroyd dεm na do, wetin if wi fכn we fכ ridyus di ACTH signal we de kכmכt frכm di bren dairekt wan?
Na de dɛn fɛn nyu target. na bin wan rεsεpכta na di bren we dεn kכl CRF1. we di kכtisol lεvεl dεm dכn, di ACTH signal de trigεr bay CRF1. Insay CAH, dis prɔses kin ambɔg.
So sayɛnsman dɛn bin tink se, if wi blok dis CRF1, wi kin ridyus di prodakshɔn fɔ ACTH, nɔto so? If dat apin, dɛn go kɔntrol di prodakshɔn fɔ andrɔjen dɛn bak, ɛn di doz fɔ stɛroyd we di pɔsin nid fɔ tek go ridyus bak.
Dis nyu aidia na in mek dɛn mek wan nyu mɛrɛsin we dɛn kɔl Crinecerfont. Dis na dכg we dεn dεn mek fכ adrεs wan fכndamεntכl prכblεm fכ CAH bay we i de blכk CRF1.
Test we dɛn kin du bifo dɛn gi pipul dɛn mɛrɛsin
Dɛn nɔ kin gi nyu mɛrɛsin to pipul dɛn jɔs afta dɛn dɔn fɛn am. I nid fɔ du sɔm tɛst fɔ no if i sef ɛn i wok. Di Crinecerfont drɔg bin go tru ɔl dɛn step ya.
1. Labכtכri εn animal tεst: di fכs step na fכ tεst di dכg pan sεl dεm εn animal dεm na di labכtכri. Dis na fɔ no if di mɛrɛsin gɛt pɔyzin ɔ if i go ambɔg di ɔgan dɛn. Jɔs imajin, na lɛk wan pan ɛvri 10,000 nyu drɔgs dɛn nɔmɔ kin pas ɔl dɛn prɔblɛm dɛn ya ɛn dɛn kin gri fɔ tek am.
2. Klinik Trayal: Afta animal tɛst dɔn wok fayn, dɛn kin du mɔtalman trial insay sɔm stej dɛn.
Aw di tɛst rizɔlt fɔ Crinecerfont de?
Di trayal dɛn we dɛn dɔn du pan dis drɔg dɔn gi rili gud rizɔlt. Ɛspɛshali di Faz II & Faz III trayal dɛn rili impɔtant.
Faz II - Di rizulyt fɔ yɔŋ pikin dɛn
Na ya, dɛn gi wan smɔl grup fɔ yɔŋ pikin dɛn (we ol 14-17 ia) we gɛt CAH di mɛrɛsin fɔ 14 dez ɛn dɛn si di chenj dɛn we de na dɛn ɔmon lɛvɛl. Di tin dɛn we bin apin bin mek wi sɔprayz.
- ACTH (bren signal) bin dכn bay 57% .
- 17-OHP (wan indikεtכ fכ andrכjen prodakshכn) dכn dכn bay 69% .
- Androstenedione (wan kayn androgen) bin dכn dכn bay 58% .
Faz III - Rizult fɔ big grup dɛn
Dɛn tin ya we dɛn bin dɔn fɛn ɛnkɔrej dɛn, sayɛnsman dɛn bin du tu big trayal dɛn we dɛn yuz big pipul ɛn pikin dɛn, usay dɛn bin gi sɔm di mɛrɛsin we dɛn kɔl Crinecerfont ɛn ɔda wan dɛn bin gi dɛn wan plesibo we tan lɛk drɔg bɔt i nɔ bin gɛt ɛni ɛfɛkt.
| Tim | Men rizulyt | Tɔk bɔt |
|---|---|---|
| Big pipul dɛn (we de tek Crinecerfont) . | Ridyus di stɛroyd doz | bay we dεn kכntrכl di כmon lεvεl, i pכsibul fכ ridyus di de doz כf stεroyd dεm bay 27% . |
| Big pipul dɛn (we tek plesibo) . | Ridyus di stɛroyd doz | Di stεroyd dכz bin כnli rεdכks bay 10%. |
| Pikin dɛn (we de tek Crinecerfont) . | Androgen lεvεl εn stεroyd dכz | afta 4 wik, di androgen lεvεl dεn dכn bכku bכku wan. We di trayal dɔn, di stɛroyd doz bin ridyus bay 18% . |
| Pikin dɛn (dɛn tek plesibo) . | Androgen lεvεl εn stεroyd dכz | di androgen lεvεl dεm bin inkrεs. di stεroyd dכz bin gεt fכ inkrεs bay lεk 6%. |
Dɛn rizɔlt ya sho klia wan se Crinecerfont de ɛp fɔ kɔntrol di androgen lɛvɛl, pan ɔl we i de ridyus di doz fɔ stɛroyd we kin ambɔg di sik pipul dɛn fɔ lɔng tɛm.
Aw dis mɛrɛsin sef?
Stɔdi dɛn dɔn sho se dis mɛrɛsin nɔ gɛt ɛni siriɔs sayd ɛfɛkt. Bɔrku pan di sayd ɛfɛkt dɛm wae dɛn kin ripɔt na smɔl smɔl tin dɛm ɛn na fɔ shɔt tɛm nɔmɔ.
- Taya
- Ed de at
- Nos we kol ɔ we stɔf
- Mɔsul ɔ bɛlɛ pen
- Pen na di jɔyn dɛn
Di wangren big wɔnin we kin kam wit dis mɛrɛsin na dat if yu nɔ tek di rayt doz fɔ stɛroyd we yu dɔktɔ tɛl yu , yu kɔtisol lɛvɛl kin go dɔŋ bad bad wan, we kin mek yu gɛt wan sik we kin mek yu layf de pan denja we dɛn kɔl adrenal insufficiency. Bɔt nɔbɔdi we bin tek di mɛrɛsin fayn fayn wan na di stɔdi dɛn nɔ bin dɔn gɛt dis sik . So, i rili impɔtant fɔ fala wetin yu dɔktɔ tɛl yu fɔ du.
Nyu op fɔ CAH tritmɛnt
Bikɔs dɛn tin ya dɔn wok fayn, di FDA (di men ɛjɛnsy we de gri fɔ tek mɛrɛsin) na Amɛrika dɔn gi spɛshal aprɔval fɔ dis mɛrɛsin. Crinecerfont (brand nem Crenessity) nɔto mɛrɛsin fɔ CAH. Bɔt na big step fɔ go bifo pan di tritmɛnt fɔ CAH.
Naw dɔktɔ dɛn kin gi yu dis mɛrɛsin apat frɔm di tritmɛnt we yu de gi yu naw wit stɛroyd (ad-on therapy) . Dis kin ɛp fɔ ridyus yu stɛroyd doz. Dis min se yu kin gɛt smɔl sayd ɛfɛkt frɔm stɛroyd, bɛtɛ kɔntrol yu ɔmon lɛvɛl, ɛn gɛt bɛtɛ kwaliti layf.
Mɛsej we dɛn kin kɛr go na os
- CAH (Congenital Adrenal Hyperplasia) na wan sik we de kɔmɔt frɔm di jɛnɛtiks we kin afɛkt di adrenal gland dɛn ɛn mek di ɔmon dɛn nɔ balans.
- Crinecerfont (Crenessity) na nyu drɔg we dɛn dɔn gri fɔ fɔ CAH, we dɛn kin gi apat frɔm sterɔyd tritmɛnt.
- dis de ridyus di hכy androjen כmon lεvεl dεm na di bכdi εn i kin εp fכ rεdכks di dכz fכ stεroyd dεm evride.
- We yu ridyus di doz fɔ stɛroyd, chans de fɔ mek yu nɔ gɛt di bad bad tin dɛn we kin apin we yu yuz stɛroyd fɔ lɔng tɛm.
- Pan ɔl we dis nɔto mɛrɛsin fɔ di sik, na big step fɔ go bifo fɔ mek di kwaliti fɔ layf fɔ di wan dɛn we gɛt CAH bɛtɛ.
- If yu ɔ yu pikin gɛt CAH, i impɔtant fɔ tɔk to yu ɛndokrinɔlɔjis ɔ famili dɔktɔ fɔ si if dis nyu tritmɛnt fayn fɔ yu .
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