Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I
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
Published in Pediatrics (Evanston) (01.01.2009)
Published in Pediatrics (Evanston) (01.01.2009)
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Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study
Trefz, Friedrich K., MD, Burton, Barbara K., MD, Longo, Nicola, MD, PhD, Casanova, Mercedes Martinez-Pardo, MD, Gruskin, Daniel J., MD, Dorenbaum, Alex, MD, Kakkis, Emil D., MD, PhD, Crombez, Eric A., MD, Grange, Dorothy K., MD, Harmatz, Paul, MD, Lipson, Mark H., MD, Milanowski, Andrzej, MD, PhD, Randolph, Linda Marie, MD, Vockley, Jerry, MD, PhD, Whitley, Chester B., MD, PhD, Wolff, Jon A., MD, Bebchuk, Judith, ScD, Christ-Schmidt, Heidi, MSE, Hennermann, Julia B., MD
Published in The Journal of pediatrics (01.05.2009)
Published in The Journal of pediatrics (01.05.2009)
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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 (laronidase)
Wraith, J Edmond, Beck, Michael, Lane, Roderick, van der Ploeg, Ans, Shapiro, Elsa, Xue, Yong, Kakkis, Emil D, Guffon, Nathalie
Published in Pediatrics (Evanston) (01.07.2007)
Published in Pediatrics (Evanston) (01.07.2007)
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Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I
Dickson, Patricia, Peinovich, Maryn, McEntee, Michael, Lester, Thomas, Le, Steven, Krieger, Aimee, Manuel, Hayden, Jabagat, Catherine, Passage, Merry, Kakkis, Emil D
Published in The Journal of clinical investigation (01.08.2008)
Published in The Journal of clinical investigation (01.08.2008)
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Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints
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
Published in Orphanet journal of rare diseases (10.02.2015)
Published in Orphanet journal of rare diseases (10.02.2015)
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A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years
Sifuentes, Monica, Doroshow, Robin, Hoft, Richard, Mason, Greg, Walot, Irwin, Diament, Michael, Okazaki, Susan, Huff, Kenneth, Cox, Gerald F., Swiedler, Stuart J., Kakkis, Emil D.
Published in Molecular genetics and metabolism (01.02.2007)
Published in Molecular genetics and metabolism (01.02.2007)
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Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)
Wraith, James E, Clarke, Lorne A, Beck, Michael, Kolodny, Edwin H, Pastores, Gregory M, Muenzer, Joseph, Rapoport, David M, Berger, Kenneth I, Swiedler, Stuart J, Kakkis, Emil D, Braakman, Tanja, Chadbourne, Elenie, Walton-Bowen, Karen, Cox, Gerald F
Published in The Journal of pediatrics (01.05.2004)
Published in The Journal of pediatrics (01.05.2004)
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Enzyme-Replacement Therapy in Mucopolysaccharidosis I
Kakkis, Emil D, Muenzer, Joseph, Tiller, George E, Waber, Lewis, Belmont, John, Passage, Merry, Izykowski, Barbara, Phillips, Jeffrey, Doroshow, Robin, Walot, Irv, Hoft, Richard, Yu, Kian Ti, Okazaki, Susie, Lewis, Dave, Lachman, Ralph, Thompson, Jerry N, Neufeld, Elizabeth F
Published in The New England journal of medicine (18.01.2001)
Published in The New England journal of medicine (18.01.2001)
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A dose-optimization trial of laronidase (Aldurazyme ®) in patients with mucopolysaccharidosis I
Giugliani, Roberto, Rojas, Verónica Muñoz, Martins, Ana Maria, Valadares, Eugênia R., Clarke, Joe T.R., Góes, José E.C., Kakkis, Emil D., Worden, Mary Alice, Sidman, Marisa, Cox, Gerald F.
Published in Molecular genetics and metabolism (2009)
Published in Molecular genetics and metabolism (2009)
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Hemodynamic changes after protamine administration: Association with mortality after coronary artery bypass surgery
WELSBY, Ian J, NEWMAN, Mark F, PHILLIPS-BUTE, Barbara, MESSIER, Robert H, KAKKIS, Emil D, STAFFORD-SMITH, Mark
Published in Anesthesiology (Philadelphia) (01.02.2005)
Published in Anesthesiology (Philadelphia) (01.02.2005)
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