Risdiplam Powder Cas No.1825352-65-5
Khoom Source: Synthetic
Product Specification: Ntau dua lossis sib npaug li 98%
CAS Nr .: 1825352-65-5
Molecular Formula: C₂₂H₂₃N₅O
Molecular Luj: 373.46 g / mol
Test Method: HPLC
Cov khoom xyaw nquag: Risdiplam
Cov yam ntxwv ntawm cov khoom: feem ntau yog dawb los tawm - cov hmoov dawb.
Ntim Cov Khoom: 1kg / hnab; 25kg / nruas
Cia Lub Sijhawm: Yuav tsum khaws cia ntev - lub sijhawm ntawm -20℃hauv qhov chaw qhuav, tiv thaiv lub teeb nyob rau hauv qhov chaw tsis muaj zog.
Lub neej txee: 24 lub hlis
Khoom Description
Risdiplam yog ib tug me me molecule, muab qhov ncauj raws li ib tug syrup. Nws yog kev ciaj sia ntawm lub cev muaj zog neuron 2 (SMN2) splicing modifier tsim los kho tus txha caj qaum atrophy (SMA) los ntawm kev nce qib ntawm kev ua haujlwm SMN protein thoob plaws hauv lub cev.
Khoom muaj nuj nqi
Lub luag haujlwm tseem ceeb ntawm Risdiplam yog txhawm rau ua kom cov khoom tiav tag nrho -ntev SMN protein los ntawm kev hloov pauv qhov sib txuas ntawm SMN2 noob. Nws tsis yog ib qho kev kho rau SMA, tab sis nws ua haujlwm los nres lossis qeeb qhov kev loj hlob ntawm tus kab mob los ntawm kev hais txog nws cov caj ces hauv paus.
Khoom Indications
Risdiplam yog qhia rau kev kho mob ntawm tus txha caj qaum atrophy (SMA) hauv cov neeg mob ntawm txhua lub hnub nyoog, txij li ob lub hlis thiab siab dua.
Qhov no suav nrog cov neeg mob ua ntej -Symptomatic SMA, Hom 1, Hom 2, thiab Hom 3.
Mechanism ntawm Action
Risdiplam ua haujlwm los ntawm kev khi rau ob qhov chaw tshwj xeeb ntawm SMN2 ua ntej-messenger RNA (pre-mRNA), cov noob uas feem ntau tsim cov SMN protein tsis ruaj khov (SMNΔ7).
Los ntawm kev khi rau cov chaw no, Risdiplam txhawb nqa kev suav nrog exon 7 thaum lub sijhawm RNA splicing txheej txheem.
Qhov kev hloov kho no ua rau muaj feem ntau dua ntawm SMN2 cov noob ua tiav -ntev, ua haujlwm tau zoo SMN protein.
Kev nce qib ntawm SMN protein txhim kho txoj sia nyob thiab kev ua haujlwm ntawm lub cev muaj zog neurons, uas yog qhov tseem ceeb rau cov leeg nqaij.
Cov khoom siv
Txoj Kev Ua Haujlwm: Qhov ncauj (kua kua dej)
Dosing: Ib hnub ib hnub, tom qab noj mov.
Kev tswj hwm: Cov koob tshuaj yog tus neeg mob- tshwj xeeb thiab nyob ntawm tus neeg mob lub hnub nyoog thiab lub cev hnyav. Nws yog muab los ntawm kev siv lub qhov ncauj syringe rau kev noj kom raug.
Qhov Zoo Tshaj Plaws: Raws li cov kua uas tuaj yeem noj hauv qhov ncauj lossis hauv lub raj mis pub mis, tshwj xeeb tshaj yog tsim rau cov menyuam mos thiab cov menyuam yaus, muab cov kev kho mob uas tsis yog -invasive piv rau kev txhaj tshuaj lossis tshuaj kho noob.
Pharmacological teebmeem
Thawj Pharmacodynamic nyhuv:
Kev txhawb nqa, koob tshuaj - nyob ntawm qhov nce hauv cov ntshav ntawm SMN protein. Qhov kev nce no tau pom nyob rau hauv lub lis piam ntawm kev pib kho thiab khaws cia thoob plaws kev kho mob
Clinical Effects:
Hauv kev sim tshuaj, kev kho mob nrog Risdiplam tau ua rau muaj kev txhim kho tseem ceeb hauv kev ua haujlwm ntawm lub cev muaj zog (piv txwv li, tswj lub taub hau, zaum, sawv, taug kev) uas tsis tau pom nyob rau hauv keeb kwm ntawm kev kho tsis tau SMA. Nws tau pom tias yuav txhim kho kev ciaj sia thiab kev tshwm sim- muaj sia nyob dawb hauv cov me nyuam mos nrog Hom 1 SMA.
Kev faib tawm:
Nws tau muab faib ua ke, txhais tau tias nws nce SMN protein tsis yog nyob rau hauv lub hauv nruab nrab lub paj hlwb, tab sis kuj nyob rau hauv peripheral cov ntaub so ntswg thiab lub cev, uas yuav muab kev kho mob dav dav.
Safety Profile:
Feem ntau cov kev phiv tshwm sim muaj xws li kub taub hau, raws plab, ua pob khaus, qhov ncauj thiab aphthous ulcers, mob pob qij txha, thiab kab mob urinary. Kev saib xyuas lub siab ua haujlwm tsis tu ncua yog pom zoo.
Cim npe nrov: Risdiplam hmoov cas no.1825352-65-5, Tuam Tshoj risdiplam hmoov cas no.1825352-65-5 manufacturers, lwm tus neeg, Hoobkas
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