MUCOPOLYSACCHARIDOSIS TYPE IIIA

Clinical trials for MUCOPOLYSACCHARIDOSIS TYPE IIIA explained in plain language.

Never miss a new study

Get alerted when new MUCOPOLYSACCHARIDOSIS TYPE IIIA trials appear

Sign up with your email to follow new studies for MUCOPOLYSACCHARIDOSIS TYPE IIIA, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Our safety recommendation!

By submitting, you agree to our Terms of use

Not yet finished but already full! 1

New hope for children battling rare brain disease

Disease control Ongoing

This early-stage study is testing an experimental drug called DNL126 in children with Sanfilippo syndrome type A, a rare and devastating genetic disorder that causes progressive brain damage. Researchers will assess the drug's safety, how it moves through the body, and look for e…

Matched conditions: MUCOPOLYSACCHARIDOSIS TYPE IIIA

Phase: PHASE1, PHASE2 • Sponsor: Denali Therapeutics Inc. • Aim: Disease control

Last updated Apr 01, 2026 14:41 UTC

MUCOPOLYSACCHARIDOSIS TYPE IIIA

Get alerted when new MUCOPOLYSACCHARIDOSIS TYPE IIIA trials appear

New hope for children battling rare brain disease