What patients and families need to know
The Children's National Research Institute
K99/R00:1K99HL130035, National Institutes of Health
Mechanisms of anti-inflammation and membrane stabilization in muscular dystrophy
Glucocorticoids are among the most prescribed drugs in the world, however they have harsh side effects and an unclear mechanism of action. This work will dissect glucocorticoid mechanisms of action to produce knowledge that leads to next-generation therapies and new standards of care for Duchenne muscular dystrophy skeletal and cardiac muscle, and other disorders of inflammation or disruptions of membrane integrity.
1U54HD090254-01, National Institutes of Health
Bridging pharmacodynamic biomarkers to clinical outcomes in pediatric inflammatory diseases
Our major goal is the validation of pharmacodynamic biomarker panels that will not only be of use for the management of patients treated chronically with glucocorticoids but also for the development of new anti-inflammatory drugs with improved efficacy and less side effects.
Foundation to Eradicate Duchenne Grant
Target-defined drug responses in mdx heart and muscle
Heart failure and steroid side effects are two very important problems for Duchenne muscular dystrophy (DMD) patients and their families. We are producing a new dissociative steroidal drug for DMD with the potential to improve both heart and skeletal muscle in DMD while avoiding side effects. Our goal is to leverage recent (samples in hand) and ongoing preclinical studies with selective drugs and receptor mutant mice into target-defined biomarkers that are predictive of improved heart and muscle health.
1L40AR068727-0- NIH Loan Repayment Award, National Institutes of Health
Mechanisms for treatment of dystrophinopathies
This NIH Award provides funding to fully repay academic loans while performing an approved research program in pediatric medical research. Research focuses on mechanisms of nuclear receptor signaling and the potential of a new ligand to treat dystrophin-deficient skeletal and heart muscle defects.
Clark Charitable Foundation Grant, Clark Charitable Foundation
Systems biology of therapeutic responses in muscular dystrophy
In orphan diseases, the decisions to move drugs from preclinical animal to human testing and to then approve drugs for clinical use are great challenges. Our goal is to use multi-level expression profiling to leverage successes in muscular dystrophy preclinical trials into a set of mechanism-defined predictive biomarkers that drive development of next generation precision medicines and combination therapies.
Rehab Pilot Project in 5R24HD050846-10
MRI life history of muscle pathology in the mdx mouse model of muscular dystrophy
The goal is to explore the dynamic muscle disease processes in mdx muscular dystrophy mice using several non-invasive MRI imaging and NMR spectroscopy technologies to establish sensitive and objective outcome measures for muscle damage and its alleviation.
Northwestern Presidential Award, Presidential Fellowship, Northwestern University
Funded by the President of the University and awarded by The Graduate School, this highly competitive award is the most prestigious fellowship awarded by Northwestern. Presidential Fellows combine outstanding intellectual ability with the capacity to be future leaders in their field.