Androgen Receptor Mediators
Aside from avoiding strenuous exercise to prevent further muscle damage there is no treatment or cure for LGMD2B/MM. Glucocorticoid (steroid) hormone-treatment suppresses inflammation and damage in other muscular dystrophies and muscle inflammatory diseases but it is not effective in reducing muscle damage in LGMD2B/MM patients and can even make it worse. Androgens are well-known stimulants of muscle development and promoters of cell survival. Using mouse and human models of muscle development we are analyzing the muscle-building mechanisms of androgen receptor agonists in the presence and absence of dysferlin. We are also establishing screens to identify SARMS (Selective androgen receptor mediators) that are preferentially muscle building with minimal side-effects as protective therapy against dysferlinopathy.