MUCOPOLYSACCHARIDOSIS II
Clinical trials for MUCOPOLYSACCHARIDOSIS II explained in plain language.
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New cell booster may help cord blood transplants reach the brain in kids with rare diseases
Disease control Recruiting nowThis early-stage study tests whether adding special cells (DUOC-01) to a standard cord blood transplant is safe for children with inherited metabolic diseases that damage the brain. About 40 participants, from 1 week to 21 years old, will receive the cells via a spinal injection.…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated May 17, 2026 01:49 UTC
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New registry aims to unlock secrets of rare lysosomal diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 people with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to learn how these conditions develop and how they appear before birth. Researchers will review medical records and test samples t…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 17, 2026 01:47 UTC
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French researchers launch largest-ever MPS patient registry to unlock disease secrets
Knowledge-focused Recruiting nowThis study will collect health information from 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers aim to better understand how the disease progresses and how it is currently managed with or without specific treatments. No new …
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Apr 29, 2026 15:03 UTC