This topic contains 0 replies, has 1 voice, and was last updated by  Michael Morale 4 weeks, 1 day ago.

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     Michael Morale 

    In this episode of the Morale Monologue, I discuss some exciting news regarding AveXis’s drug candidate ZOLGENSMA. We have received information that AveXis has provided additional information to the FDA, and have asked the FDA to broaden the label of their potential new drug therapy. The initial request was for SMA Type 1 infants, who were 9 months of age or younger. Now, AveXis is requesting that the label also include children with SMA Type 2, who are 5 years of age or younger, based on some of the results behind the trial of their intrathecal (IT) delivery method.


    Web Link To AveXis Article:


    Are you interested in understanding gene therapy? has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases.



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