Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease-;about a half million people in the United States alone suffer from the condition.
Results support the development of PXL770 in a Phase 2 clinical program for ADPKD Regulatory News: POXEL SA (Paris:POXEL) (Euronext: POXEL FR0012432516), a clinical stage biopharmaceutical company
Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in the peer-reviewed journal Human Gene Therapy.