Raheel Shafi

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

Raheel Shafi, Sai Prasad N. Iyer, Lesley G. Ellies et al. · Proceedings of the National Academy of Sciences · 2000 · 795 citations

Life Sciences, Genetics, Molecular Biology +15

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study

Loren Peña, Richard J. Barohn, Barry J. Byrne et al. · Neuromuscular Disorders · 2018 · 90 citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +12

Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study

Ans van der Ploeg, Pierre G. Carlier, Robert-Yves Carlier et al. · Molecular Genetics and Metabolism · 2016 · 78 citations · Full text

Medicine, Biochemistry, Internal Medicine +17

Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study

Si Houn Hahn, David Kronn, Nancy D. Leslie et al. · Genetics in Medicine · 2018 · 31 citations

Pharmacy, Pediatrics, Pharmacotherapy +13

Pathogenesis of the B variant of encephalomyocarditis virus

Raheel Shafi, D. Roselyn Cerutis, David Giron · Journal of Medical Virology · 1993 · 13 citations

Life Sciences, Infectious Diseases, Microbiology +17