SPARK THERAPEUTICS GENE THERAPYA developing treatment that aims to fix, replace, or add genetic instructions. RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapy

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. research. This

CRANBURY, N.J., Sept. 10, 2018—Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare

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

The AMDA is excited to announce that the 2017 PCMA of Texas Pull for Pompe will take place on Saturday,

The seventh annual AMDA Research Grant (the 2016 Helen Walker Research Grant) was awarded to Dr. Frédéric Lofaso with the