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The Children's Research Institute

Chris Heier, Ph.D., Laboratory

predisone

We have several funded projects which focus on steroid signaling and gene expression in Duchenne muscular dystrophy (DMD) and other chronic inflammatory diseases.

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About Our Lab

Our goal is to develop next generation treatments and biomarkers for diseases in children. Our particular focus is on genetic diseases of muscle (such as Duchenne muscular dystrophy), but by targeting the underlying molecular biology of disease we believe we can help a broad array of human illnesses. For example, we are currently dissecting molecular pathways involved in steroid signaling. This has helped to develop a new anti-inflammatory drug named vamorolone (previously named VBP15) to the point that it is now in clinical trials for Duchenne muscular dystrophy. Early work indicates this drug has the potential to replace prednisone as the standard of care for muscular dystrophy, by providing a drug that selectively targets the same receptors to be more effective while also being much safer. Through our understanding of drug pathways, we are also developing serum-based biomarkers reflective of drug activities in patients. Ultimately, we feel this approach has the power to greatly improve the treatment of a large number of diseases featuring chronic inflammation.

Chris Heier, Ph.D., Laboratory

Chris Heier

Our goal is to develop next generation treatments and biomarkers for diseases in children.