Innovations in steroid chemistry, pharmacodynamic biomarkers, clinical trial design, and business models for orphan drug development
Clinical Trials
Vamorolone is in clinical testing for DMD, with 46 patients enrolled in VBP15-LTE (NCT03038399) and recruitment of 120 new patients initiated for VBP15-004 (NCT03439670).
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Research
microRNAs shown to define dystrophin levels in patient muscle (Fiorillo et al. 2015) – IP licensed to ReveraGen.
