Spark Therapeutics Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. More Research Webinar Download an audio only version

Spark Therapeutics Announces New Preclinical Data for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. More Candidate Data from IND-enabling studies in three species support

SPARK THERAPEUTICS GENE THERAPYA developing treatment that aims to fix, replace, or add genetic instructions. More 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 tha... More 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 DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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