Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

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

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Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science