Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

Abstract Background Mucopolysaccharidosis type III is a progressive, neurodegenerative lysosomal storage disorder for which there is currently no effective therapy. Though numerous potential therapies are in development, there are several challenges to conducting clinical research in this area. We s...

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Bibliographic Details
Main Authors: Arunabha Ghosh, Elsa Shapiro, Stewart Rust, Kathleen Delaney, Samantha Parker, Adam J Shaywitz, Adelaida Morte, Gillian Bubb, Maureen Cleary, Tien Bo, Christine Lavery, Brian W Bigger, Simon A Jones
Format: Article
Language:English
Published: BMC 2017-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Lysosomal storage disorders
mucopolysaccharidoses
Mucopolysaccharidosis Type III
Sanfilippo syndrome
Clinical trials
Natural history
Online Access:http://link.springer.com/article/10.1186/s13023-017-0675-4

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

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