Docket Number:
FDA-2019-D-5404
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research
Center for Drug Evaluation and Research

The purpose of this guidance is to provide recommendations to sponsors regarding eligibility criteria, trial design considerations, and efficacy endpoints to enhance clinical trial data quality and foster greater efficiency in development programs for drugs to treat mucopolysaccharidosis type III (MPS III; also called Sanfilippo syndrome).

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