Other examples include obesity, Gaucher's disease, phenylketonuria (PKU), medium-chain acyl-CoA dehydrogenase deficiency (MCADD), maple syrup urine disease (MSUD), and hemochromatosis. These ...
(“Next Generation Gene Therapeutics” or “NGGT”), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced positive new data ...