Researchers from the Erasmus MC in Rotterdam, Netherlands, have succeeded in generating muscle cells from skin cells. This is significant

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This

CONCORD, Mass., July 11, 2017—Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic

he AMDA was saddened to learn of the sudden passing of Henri Termeer, former CEO of Genzyme. We have known

Excerpt . . . . CRANBURY, N.J., May 15, 2017 —Amicus Therapeutics today announced positive functional data from initial patients