Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III-Reference-Cited by-同舟云学术

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

Published:2017-06-26 Issue:1 Volume:12 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Ghosh Arunabha,Shapiro Elsa,Rust Stewart,Delaney Kathleen,Parker Samantha,Shaywitz Adam J,Morte Adelaida,Bubb Gillian,Cleary Maureen,Bo Tien,Lavery Christine,Bigger Brian W,Jones Simon A

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-017-0675-4.pdf

Reference108 articles.

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3. Baehner F, Schmiedeskamp C, Krummenauer F, Miebach E, Bajbouj M, Whybra C, et al. Cumulative incidence rates of the mucopolysaccharidoses in Germany. J Inherit Metab Dis. 2005;28:1011–7.

4. Poorthuis BJ, Wevers RA, Kleijer WJ, Groener JE, de Jong JG, van Weely S, et al. The frequency of lysosomal storage diseases in The Netherlands. Hum Genet. 1999;105:151–6.

5. Heron B, Mikaeloff Y, Froissart R, Caridade G, Maire I, Caillaud C, et al. Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece. Am J Med Genet A. 2011;155a:58–68.

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