Moris Danon News Today

— Continued to advance clinical gene therapy programs for the treatment of Danon Disease, Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) —— Updates on all four programs to be presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGC...

It’s the last day in February, and that means it’s time to shine a light on rare disease—literally, in the case of "Light up for Rare." Drugmakers such as BioMarin are illuminating everything from factories to Times Square to raise awareness.