Tamoxifen is a selective estrogen receptor modulator (SERM) used to treat estrogen-dependent breast cancer, as well as some hormonal disorders and has been given to some pre-pubescent boys. In breast tissue the drug blocks estrogen receptors and slows the development of cancer. In other tissues the drug may stimulate estrogen receptors instead of blocking them. Data from animals shows that tamoxifen can trigger substantial improvements in muscle strength in mice that lack dystrophin. How the drug works in the mice is not yet understood.
A Phase 3 clinical trial has been completed.
This program is sponsored by University Hospital, Basel, Switzerland. Preclinical work was partially funded by Parent Project Muscular Dystrophy.