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	1.	Enzyme replacement therapy for the mucopolysaccharide storage disorders. [electronic resource] by Kakkis, Emil D Producer: 20021104 In: Expert opinion on investigational drugs vol. 11 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	2.	Patients as key partners in rare disease drug development. [electronic resource] by Bronstein, Max G Kakkis, Emil D Producer: 20170215 In: Nature reviews. Drug discovery vol. 15 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	3.	The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases. [electronic resource] by Miyamoto, Brigitta E Kakkis, Emil D Producer: 20111121 In: Orphanet journal of rare diseases vol. 6 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	4.	Accessing the accelerated approval pathway for rare disease therapeutics. [electronic resource] by Kakkis, Emil D Kowalcyk, Sara Bronstein, Max G Producer: 20160823 In: Nature biotechnology vol. 34 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	5.	First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient. [electronic resource] by Fox, Joyce E Volpe, Linda Bullaro, Josephine Kakkis, Emil D Sly, William S Producer: 20151028 In: Molecular genetics and metabolism vol. 114 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	6.	Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I. [electronic resource] by Kakavanos, Revecca Turner, Chris T Hopwood, John J Kakkis, Emil D Brooks, Doug A Producer: 20030529 In: Lancet (London, England) vol. 361 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	7.	Hemodynamic changes after protamine administration: association with mortality after coronary artery bypass surgery. [electronic resource] by Welsby, Ian J Newman, Mark F Phillips-Bute, Barbara Messier, Robert H Kakkis, Emil D Stafford-Smith, Mark Producer: 20050224 In: Anesthesiology vol. 102 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	8.	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). [electronic resource] by Wraith, J Edmond Beck, Michael Lane, Roderick van der Ploeg, Ans Shapiro, Elsa Xue, Yong Kakkis, Emil D Guffon, Nathalie Producer: 20070803 In: Pediatrics vol. 120 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	9.	Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I. [electronic resource] by Dickson, Patricia Peinovich, Maryn McEntee, Michael Lester, Thomas Le, Steven Krieger, Aimee Manuel, Hayden Jabagat, Catherine Passage, Merry Kakkis, Emil D Producer: 20080925 In: The Journal of clinical investigation vol. 118 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	10.	Arterial pathology in canine mucopolysaccharidosis-I and response to therapy. [electronic resource] by Lyons, Jeremiah A Dickson, Patricia I Wall, Jonathan S Passage, Merry B Ellinwood, N Matthew Kakkis, Emil D McEntee, Michael F Producer: 20110629 In: Laboratory investigation; a journal of technical methods and pathology vol. 91 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	11.	Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints. [electronic resource] by 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 Producer: 20160121 In: Orphanet journal of rare diseases vol. 10 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	12.	A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. [electronic resource] by 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 Producer: 20070329 In: Molecular genetics and metabolism vol. 90 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	13.	A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. [electronic resource] by 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 Producer: 20090121 In: Molecular genetics and metabolism vol. 96 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	14.	Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs. [electronic resource] by Vera, Moin Lester, Thomas Zhao, Bin Tiger, Pascale Clarke, Scott Tippin, Brigette L Passage, Merry B Le, Steven Q Femenia, Javier Lemontt, Jeffrey F Kakkis, Emil D Dickson, Patricia I Producer: 20130225 In: JIMD reports vol. 8 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	15.	Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. [electronic resource] by 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 Producer: 20090206 In: Pediatrics vol. 123 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	16.	Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). [electronic resource] by 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 Producer: 20040603 In: The Journal of pediatrics vol. 144 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	17.	Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. [electronic resource] by Trefz, Friedrich K Burton, Barbara K Longo, Nicola Casanova, Mercedes Martinez-Pardo Gruskin, Daniel J Dorenbaum, Alex Kakkis, Emil D Crombez, Eric A Grange, Dorothy K Harmatz, Paul Lipson, Mark H Milanowski, Andrzej Randolph, Linda Marie Vockley, Jerry Whitley, Chester B Wolff, Jon A Bebchuk, Judith Christ-Schmidt, Heidi Hennermann, Julia B Producer: 20090505 In: The Journal of pediatrics vol. 154 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)