Khan Nedd

Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat

Dominique P. Germain, Derralynn Hughes, Daniel G. Bichet et al. · New England Journal of Medicine · 2016 · 556 citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +17

Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study

Derralynn Hughes, Suma P. Shankar, Gere Sunder–Plassmann et al. · Journal of Medical Genetics · 2016 · 386 citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +17

Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study

Ulla Feldt‐Rasmussen, Derralynn Hughes, Gere Sunder‐Plassmann et al. · Molecular Genetics and Metabolism · 2020 · 77 citations · Full text

Pharmacy, Pharmacology, Pharmacotherapy +13

Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease

Laura E. Case, Carl Bjartmar, Claire Morgan et al. · Neuromuscular Disorders · 2014 · 59 citations · Full text

Life Sciences, Internal Medicine, Pharmacotherapy +10

Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study

Eric Wallace, Özlem Göker-Alpan, William R. Wilcox et al. · Journal of Medical Genetics · 2023 · 43 citations · Full text

Medicine, Nephrology, Pharmacotherapy +17