NEJM小分子药物Risdiplam可增
2021-12-29 来源：不详 浏览次数：次Abstract
Background:Type1spinalmuscularatrophyisarare,progressiveneuromusculardiseasethatiscausedbylowlevelsoffunctionalsurvivalofmotorneuron(SMN)protein.Risdiplamisanorallyadministered,smallmoleculethatmodifiesSMN2pre-messengerRNAsplicingandincreaseslevelsoffunctionalSMNprotein.
Methods:Wereporttheresultsofpart1ofatwo-part,phase2-3,open-labelstudyofrisdiplamininfants1to7monthsofagewhohadtype1spinalmuscularatrophy,whichischaracterizedbytheinfantnotattainingtheabilitytositwithoutsupport.Primaryout
