Innovations in steroid chemistry, pharmacodynamic biomarkers, clinical trial design, and business models for orphan drug development
Phase 1 biomarker data shows improvement of safety profiles of vamorolone over prednisolone for adrenal suppression, bone turnover, insulin resistance, and immune suppression. Learn more >
Vamorolone enrollment in Phase 2a opens at Duke University; first DMD subjects enrolled.
microRNAs shown to define dystrophin levels in patient muscle (Fiorillo et al. 2015) – IP licensed to ReveraGen.
Vamorolone, a novel compound for the treatment of Duchenne Muscular Dystrophy, holds potential to better preserve muscle function and prolong ambulation for the patient, without some of the side effects associated with glucocorticoid therapy.
ALLSCHWIL, SWITZERLAND, and ROCKVILLE (MD), USA