Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference131 articles.
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3. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome);Muenzer;Genet. Med.,2006
4. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome;Muenzer;Genet. Med.,2011
5. Long-term outcome of Hurler syndrome following bone marrow transplantation;Whitley;Am. J. Med. Genet.,1996
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