MUCOPOLYSACCHARIDOSIS II
Clinical trials for MUCOPOLYSACCHARIDOSIS II explained in plain language.
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New hope for kids with devastating brain diseases: experimental cell therapy aims to halt progression
Disease control Recruiting nowThis early-stage study is testing the safety of adding a special type of cell, derived from umbilical cord blood, to a standard cord blood transplant for children with rare inherited metabolic diseases that damage the brain. The goal is to see if delivering these cells directly i…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Massive study launched to map rare disease journey
Knowledge-focused Recruiting nowThis study aims to gather detailed health information from 1000 French patients with a group of rare genetic disorders called Mucopolysaccharidosis (MPS). Researchers will look back at past medical records and follow patients forward to understand how these diseases naturally pro…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC