Your search returned 37 results.

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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.	Urinary glycosaminoglycans as a potential biomarker for evaluating treatment efficacy in subjects with mucopolysaccharidoses. [electronic resource] by Kakkis, Emil Marsden, Deborah Producer: 20210208 In: Molecular genetics and metabolism vol. 130 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	3.	The 21st Century Cures Act. [electronic resource] by Kakkis, Emil Bronstein, Max G Producer: 20151022 In: The New England journal of medicine vol. 373 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	4.	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)
	5.	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)
	6.	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)
	7.	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)
	8.	Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment--A retrospective chart review. [electronic resource] by Vockley, Jerry Marsden, Deborah McCracken, Elizabeth DeWard, Stephanie Barone, Amanda Hsu, Kristen Kakkis, Emil Producer: 20160706 In: Molecular genetics and metabolism vol. 116 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	9.	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)
	10.	A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease. [electronic resource] by Harmatz, Paul Whitley, Chester B Wang, Raymond Y Bauer, Mislen Song, Wenjie Haller, Christine Kakkis, Emil Producer: 20190708 In: Molecular genetics and metabolism vol. 123 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	11.	Substantial deficiency of free sialic acid in muscles of patients with GNE myopathy and in a mouse model. [electronic resource] by Chan, Yiumo Michael Lee, Paul Jungles, Steve Morris, Gabrielle Cadaoas, Jaclyn Skrinar, Alison Vellard, Michel Kakkis, Emil Producer: 20170825 In: PloS one vol. 12 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	12.	Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. [electronic resource] by Dickson, Patricia McEntee, Michael Vogler, Carole Le, Steven Levy, Beth Peinovich, Maryn Hanson, Stephen Passage, Merry Kakkis, Emil Producer: 20070725 In: Molecular genetics and metabolism vol. 91 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	13.	Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice. [electronic resource] by Dvorak-Ewell, Melita Wendt, Dan Hague, Chuck Christianson, Terri Koppaka, Vish Crippen, Danielle Kakkis, Emil Vellard, Michel Producer: 20101104 In: PloS one vol. 5 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	14.	Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII. [electronic resource] by Vogler, Carole Levy, Beth Grubb, Jeffrey H Galvin, Nancy Tan, Yun Kakkis, Emil Pavloff, Nadine Sly, William S Producer: 20060420 In: Proceedings of the National Academy of Sciences of the United States of America vol. 102 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	15.	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)
	16.	Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII. [electronic resource] by Cadaoas, Jaclyn Boyle, Gabrielle Jungles, Steven Cullen, Sean Vellard, Michel Grubb, Jeffrey H Jurecka, Agnieszka Sly, William Kakkis, Emil Producer: 20210208 In: Molecular genetics and metabolism vol. 130 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	17.	Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen. [electronic resource] by Auclair, Dyane Finnie, John White, Joleen Nielsen, Timothy Fuller, Maria Kakkis, Emil Cheng, Alphonsus O'Neill, Charles A Hopwood, John J Producer: 20100503 In: Molecular genetics and metabolism vol. 99 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	18.	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)
	19.	Aceneuramic Acid Extended Release Administration Maintains Upper Limb Muscle Strength in a 48-week Study of Subjects with GNE Myopathy: Results from a Phase 2, Randomized, Controlled Study. [electronic resource] by Argov, Zohar Caraco, Yoseph Lau, Heather Pestronk, Alan Shieh, Perry B Skrinar, Alison Koutsoukos, Tony Ahmed, Ruhi Martinisi, Julia Kakkis, Emil Producer: 20180315 In: Journal of neuromuscular diseases vol. 3 Online resources: Available from publisher's website Availability: No items available. Save to lists Add to cart (remove)
	20.	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)

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Your search returned 37 results.