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Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

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 seek to m...

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Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Position Statement
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5485703/ https://www.ncbi.nlm.nih.gov/pubmed/28651568 http://dx.doi.org/10.1186/s13023-017-0675-4

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