Author:
Qi Yulan,McKeever Kathleen,Taylor Julie,Haller Christine,Song Wenjie,Jones Simon A.,Shi Jack
Funder
Ultragenyx Pharmaceutical Inc.
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Pharmacology
Reference24 articles.
1. Neufeld E, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The metabolic and molecular bases of inherited disease. New York: McGraw-Hill; 2001.
2. Sly WS, Quinton BA, McAlister WH, Rimoin DL. Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis. J Pediatr. 1973;82:249–57.
3. Montano AM, Lock-Hock N, Steiner RD, Graham BH, Szlago M, Greenstein R, et al. Clinical course of sly syndrome (mucopolysaccharidosis type VII). J Med Genet. 2016;53:403–18.
4. Harmatz P, Giugliani R, Schwartz I, Guffon N, Teles EL, Miranda MC, et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006;148:533–9.
5. Kakkis ED, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344:182–8.
Cited by
19 articles.
订阅此论文施引文献
订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献