Raheel Shafi

The O-GlcNAc transferase gene resides on the X chromosome and is essential for embryonic stem cell viability and mouse ontogeny

Proceedings of the National Academy of Sciences of the United States of America · 2000 · First of 8 authors · 659 citations

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and…

Neuromuscular Disorders · 2019 · 75 citations