MUCOPOLYSACCHARIDOSIS I (MPS I)

Clinical trials for MUCOPOLYSACCHARIDOSIS I (MPS I) explained in plain language.

Never miss a new study

Get alerted when new MUCOPOLYSACCHARIDOSIS I (MPS I) trials appear

Sign up with your email to follow new studies for MUCOPOLYSACCHARIDOSIS I (MPS I), 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

Completed 1

Massive database tracks rare disease patients worldwide

Knowledge-focused Completed

This study created a large international registry to track people with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. It followed 1,406 patients to better understand how the disease naturally progresses and how treatments like enzyme replacement therapy work over the l…

Matched conditions: MUCOPOLYSACCHARIDOSIS I (MPS I)

Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused

Last updated Apr 01, 2026 14:43 UTC

MUCOPOLYSACCHARIDOSIS I (MPS I)

Get alerted when new MUCOPOLYSACCHARIDOSIS I (MPS I) trials appear

Massive database tracks rare disease patients worldwide