The Food and Drug Administration (FDA) has accepted for review the resubmitted Biologics License Application (BLA) for prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis ...
aDepartment of Neurology, David Geffen School of Medicine at UCLA, University of California at Los Angeles, Los Angeles, USA bUCLA Ataxia Center, University of California at Los Angeles, Los Angeles, ...
Data from Eloxx's proof-of-concept trial in patients with nonsense mutation Alport Syndrome (NMAS) presented in late-breaking presentation at America ...