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 26, 2026 05:42 UTC
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New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects health information from up to 250 people with lysosomal storage diseases, including pregnant women whose fetus has a diagnosis. Researchers will track symptoms, lab results, and development over time to better understand how these rare conditions progress. The…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 26, 2026 05:47 UTC
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French study aims to map rare MPS diseases in 1000 patients
Knowledge-focused Recruiting nowThis study is collecting health information from 1000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will look at medical records and test results to better understand how the diseases progress and how current treatments affect p…
Matched conditions: MUCOPOLYSACCHARIDOSIS II
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated May 26, 2026 05:42 UTC