TAMDMD

Duchenne muscular dystrophy (DMD) is a rare disease that affects about 1 in 3500 to 1 in 6000 boys. Patients suffer from progressive muscle wasting, respiratory and cardiac impairments and premature death. Currently, only symptomatic treatment with glucocorticoids is available; these have limited efficacy but many adverse effects. Using the mouse DMD model, our partners in Geneva, Dorchies et al., have shown that tamoxifen (TAM), given orally for periods of 2 or 15 month at doses as low as 0.3mg/kg/day, resulted in almost full recovery of force and structure of muscles. Our aim is to investigate whether TAM treatment, compared to placebo, reduces the disease progression in DMD patients. We plan a 48-week placebo controlled randomised clinical trial with 65 ambulant (6.5-10 year old) DMD patients (under stable standard treatment of care with glucocorticoids). Patients will receive 10mg (0.3mg/kg) of TAM or of a placebo daily.