Généthon is delighted with the treatment of a first patient as part of a gene therapy trial for the late form of Pompe disease, led by the company SparkTherapeutics and integrating technologies developed at Généthon
Written by Généthon | February 10, 2021 Pompe disease is an inherited genetic disorder linked to alpha-deficiencyglucosidase acid (GAA) which causes glycogen to build up in lysosomes, causing cell damage …