UA NTEJ PUAS HEMOPHILIA TUS NEEG MOB TAU TXAIS GENE THERAPY TXIJ FDA POM ZOO
TAM SIM NTAWD TSO TAWM TAM SIM NTAWD
MAY 20, 2023
NTSUAB BAY, WI-Wisconsin nyob hauv yog thawj puas hemophilia B tus neeg mob tau txais kev kho mob vim FDA pom zoo.
"Kuv xav tias honored yuav tus thawj neeg koom rau hauv txoj kev kho no sab nraum lub tsev hais plaub mus sib hais. Kuv pog tias kuv yuav tau pab los ua pov thawj tias qhov no yog ib txoj kev kho rau cov neeg uas hemophilia B." -Mason Buxton
HEMGENIX (etranacogene dezaparvovec), manufactured los uniQure Inc. thiab faib los ntawm CSL Behring LLC, yog FDA pom zoo kaum ib hlis ntawm 2022 thiab yog rau cov neeg laus uas Hemophilia B (Factor IX Deficiency) uas tam sim no siv factor IX tiv thaiv txoj kev kho, los yog muaj tam sim no los yog keeb kwm tuag hemorrhage, los yog muaj dua, spontaneous los ntshav rov.
Hemophilia B yog ib tug genetic los ntshav disorder uas ploj lawm los ntawm cov ntshav uas ploj lawm los yog insufficient theem ntawm cov ntshav clotting Factor IX, ib protein ntau yuav tsum tau ua ntshav clots kom txhob los ntshav. Cov tsos mob yuav muaj xws li ntev los yog hnyav los ntshav tom qab raug mob, phais, los sis kho hniav; Rau cov neeg mob hnyav, los ntshav rov yuav tshwm sim spontaneously tsis muaj tseeb ua kom muaj tseeb. Prolonged los ntshav rov yuav ua rau muaj teeb meem loj, xws li los ntshav rau hauv pob qij txha, nqaij los yog hauv tej yam kabmob, xws li lub paj hlwb. (Commissioner, O. ntawm tus (2022) FDA pom zoo thawj noob rau cov neeg laus uas hemophilia B, U.S. Zaub mov thiab tshuaj Administration. Muaj nyob rau: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b#:~:text=Today%2C%20the%20U.S.%20Food%20and,current%20or%20historical%20life%2Dthreatening (Accessed: 23 Lub Tsib Hlis Ntuj 2023).
Txawm hais tias HEMGENIX tsis yog ib tug kho rau Hemophilia B cov neeg mob, cov kev kho mob yuav txo tau, yog hais tias tsis yog, eliminate txhua lub limtiam tiv thaiv tau hloov thiab los ntshav.
"HEMGENIX yog ib qho tseem ceeb ntxiv rau cov kev kho tam sim no paradigm thiab ib long-awaited advancement rau lub hemophilia B zej zog," hais Matthew Ryan, MD, neeg laus Hematologist thiab kev kho mob thawj coj ntawm Hemophilia Outreach Center, Ntsuab Bay, WI. "Qhov no exciting kho muaj cov neeg mob uas yuav nyob ib lub neej tsis nquag bleeds los yog tus tab kaum ntawm kev mus tsis tu ncua. Muaj cov tshiab ib-lub sij hawm kho kuj sparks ntxiv sib tham hauv lub zej zos thaum nws tawm los kho cov kev xaiv thiab hom phiaj, uas yog ib qho tseem ceeb heev thaum tswj ib lub neej-ntev mob, raws li ib tug neeg yuav tsum tau hloov rau ntau yam."
"Kuv cia li pog tias cov neeg mob muaj no caij nyoog tshiab uas yuav ua rau tej lub neej zoo versus lawv prophylaxis kho! Nyob ib lub neej dawb los recurrent prophylaxis, tsis muaj ntawd ntxhov siab vim yog hais tias lawv yuav muaj ib tug bleed los yog hais tias lawv muaj qhov zoo tshaj yuav nrog lawv, tsis worrying txog kev puas tsuaj rau lawv ob leeg los ntawm breakthrough bleeds....nws yuav lifechanging rau lawv." - Andrea Miller, APNP, Hemophilia Outreach Center.
