The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases von Tandon, P. K., Kakkis, Emil D.

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Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I von Clarke, Lorne A, Wraith, J. Edmond, Beck, Michael, Kolodny, Edwin H, Pastores, Gregory M, Muenzer, Joseph, Rapoport, David M, Berger, Kenneth I, Sidman, Marisa, Kakkis, Emil D, Cox, Gerald F

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The transformation of drug development for the 21st century: Time for a change von Kakkis, Emil D.

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The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases von Miyamoto, Brigitta E, Kakkis, Emil D

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Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I von Dickson, Patricia, Peinovich, Maryn, McEntee, Michael, Lester, Thomas, Le, Steven, Krieger, Aimee, Manuel, Hayden, Jabagat, Catherine, Passage, Merry, Kakkis, Emil D

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Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints von Kakkis, Emil D, O'Donovan, Mary, Cox, Gerald, Hayes, Mark, Goodsaid, Federico, Tandon, P K, Furlong, Pat, Boynton, Susan, Bozic, Mladen, Orfali, May, Thornton, Mark

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Patients as key partners in rare disease drug development von Bronstein, Max G., Kakkis, Emil D.

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Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I von Kakavanos, Revecca, Turner, Chris T, Hopwood, John J, Kakkis, Emil D, Brooks, Doug A

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Enzyme replacement therapy for the mucopolysaccharide storage disorders von Kakkis, Emil

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Enzyme replacement in a canine model of Hurler syndrome von Shull, R.M. (University of Tennessee, Knoxville, TN.), Kakkis, E.D, McEntee, M.F, Kania, S.A, Jonas, A.J, Neufeld, E.F

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Accessing the accelerated approval pathway for rare disease therapeutics: improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clin... von Kakkis, Emil D, Kowalcyk, Sara, Bronstein, Max G

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Recombinant human β-glucuronidase enzyme replacement therapy for mucopolysaccharidosis type VII: report of the first patient treated von Fox, Joyce E., Kakkis, Emil D., Sly, William S.

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Biotech leaders call for free press von Cohen, Ron, Holtzman, Steven, Levin, Jeremy M, Maraganore, John M

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FREMFØRING AF TERAPEUTISKE FORBINDELSER TIL HJERNEN OG ANDRE VÆV von KAKKIS, Emil, D

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Delivery of therapeutic compounds to the brain and other tissues von Kakkis, Emil D

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Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human {alpha}-L-Iduronidase (L... von Wraith, J. Edmond, Beck, Michael, Lane, Roderick, van der Ploeg, Ans, Shapiro, Elsa, Xue, Yong, Kakkis, Emil D, Guffon, Nathalie

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Methods for treating tumor-induced osteomalacia von San Martin, Javier, Kakkis, Emil D, Sudo, Tomohiro

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Methods and formulations for treating sialic acid deficiencies von Kakkis, Emil D

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Hemodynamic Changes after Protamine Administration von Welsby, Ian J., Newman, Mark F., Phillips-Bute, Barbara, Messier, Robert H., Kakkis, Emil D., Stafford-Smith, Mark

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EFFECTIVE AND EFFICIENT CONTROL OF SERUM PHOSPHATE FOR OPTIMAL BONE FORMATION von KAKKIS, Emil D, SAN MARTIN, Javier, SUDO, Tomohiro

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