MUCOPOLYSACCHARIDOSIS TYPE I

Clinical trials for MUCOPOLYSACCHARIDOSIS TYPE I explained in plain language.

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Scientists track rare disease drug in tiniest patients

Disease control Ongoing

This study aims to understand how a drug called laronidase works in very young children with a rare genetic disorder (MPS I) who are getting a stem cell transplant. Researchers will observe and measure the drug levels in the blood before and after the transplant. The children wil…

Matched conditions: MUCOPOLYSACCHARIDOSIS TYPE I

Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control

Last updated Mar 20, 2026 14:47 UTC

MUCOPOLYSACCHARIDOSIS TYPE I

Get alerted when new MUCOPOLYSACCHARIDOSIS TYPE I trials appear

Scientists track rare disease drug in tiniest patients