> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Sanfilippo Syndrome Drug Development Guidance
- URL: https://www.fdaweb.com/sanfilippo-syndrome-drug-development-guidance/
- Published: 2020-02-04T12:00:00.000Z
- Updated: 2026-09-14T16:01:41.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5146004

FDA has issued a draft guidance, *Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Developing Drugs for Treatment*, to make recommendations to sponsors about eligibility criteria, trial design considerations, and efficacy endpoints to enhance clinical trial data quality and foster greater efficiency in developmental programs for drugs to treat Sanfilippo syndrome. The [document](https://www.fda.gov/media/134893/download?ref=fdaweb.com) includes Introduction, Background, Important Considerations for Clinical Trials, and References.