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Abstract

Background:Type1spinalmuscularatrophyisarare,progressiveneuromusculardiseasethatiscausedbylowlevelsoffunctionalsurvivalofmotorneuron(SMN)protein.Risdiplamisanorallyadministered,smallmoleculethatmodifiesSMN2pre-messengerRNAsplicingandincreaseslevelsoffunctionalSMNprotein.

Methods:Wereporttheresultsofpart1ofatwo-part,phase2-3,open-labelstudyofrisdiplamininfants1to7monthsofagewhohadtype1spinalmuscularatrophy,whichischaracterizedbytheinfantnotattainingtheabilitytositwithoutsupport.Primaryout



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