Welcome to the
at Johns Hopkins University
School of Medicine
The long term goal of our work is to develop treatments for neurodegenerative disorders of motor neurons and peripheral nerves. Spinal muscular atrophies (SMAs) and the various forms of Charcot-Marie-Tooth disease (CMT) are among the most common inherited neurological disorders. They cause progressive muscle weakness, sensory loss, and often, early mortality. Currently, there are few disease-modifying therapies to offer patients.
By integrating studies of humans, mice, flies, and cell models our aims are to: 1) characterize the clinical, genetic, and pathological features of these debilitating disorders, 2) define molecular and cellular mechanisms of disease pathogenesis, and 3) advance novel therapeutic strategies.
To learn more about SMA and our research efforts, click here.
To learn more about CMTs and our research efforts, click here.
To see a full list of the lab's publications, click here.