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mucopolysaccharidosis VI treatment Naglazyme (galsulfase), and Palynziq (pegvaliase) for phenylketonuria. Sabry is stepping into a position vacated by the departure of Brinda Balakrishnan ...

Although great efforts have been made to address the bioavailability and tissue targeting of drugs, little is known about drug delivery to specific subcellular compartments that harbour the drug ...

First rare disease candidates to benefit from the FDA START programme are from Denali Therapeutics, Neurogene, Larimar Therapeutics, and Grace Science ...

Michael Railey, M.D., held various roles during his time at Saint Louis University School of Medicine. Before assuming the position of professor emeritus, he served as the associate dean in the Office ...