Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction

Genetics in Medicine, Published online: 25 January 2021; doi:10.1038/s41436-020-01080-yTransforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction

Source: Genetics in Medicine - January 25, 2021 Category: Genetics & Stem Cells Authors: Cindy Li Ankit K. Desai Punita Gupta Katherine Dempsey Vikas Bhambhani Robert J. Hopkin Can Ficicioglu Pranoot Tanpaiboon William J. Craigen Amy S. Rosenberg Priya S. Kishnani Source Type: research