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 Bigger, Simon Jones

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Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science