Emil Kakkis

Enzyme-Replacement Therapy in Mucopolysaccharidosis I

Emil Kakkis, Joseph Muenzer, George E. Tiller et al. · New England Journal of Medicine · 2001 · 675 citations · Full text

Life Sciences, Biochemistry, Genetics +11

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)

J. E. Wraith, L. Clarke, Michael Beck et al. · The Journal of Pediatrics · 2004 · 546 citations

Life Sciences, Biochemistry, Enzymology +7

Burosumab Therapy in Children with X-Linked Hypophosphatemia

Thomas O. Carpenter, Michael P. Whyte, Erik A. Imel et al. · New England Journal of Medicine · 2018 · 473 citations · Full text

Medicine, Endocrinology, Nephrology +17

Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study

Paul Harmatz, Roberto Giugliani, Ida Vanessa Döederlein Schwartz et al. · The Journal of Pediatrics · 2006 · 364 citations

Life Sciences, Biochemistry, Pharmacotherapy +7

Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

L. Clarke, J. E. Wraith, Michael Beck et al. · PEDIATRICS · 2008 · 346 citations

Pharmacy, Pharmacotherapy, Biopharmaceutics +12