Emil D Kakkis

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

Joseph Muenzer, Lorne A Clarke, Michael Beck, 2004, The Journal of pediatrics.

Accessing the accelerated approval pathway for rare disease therapeutics

Emil D Kakkis, Sara Kowalcyk, Max G Bronstein, 2016, Nature Biotechnology.