MUCOPOLYSACCHARIDOSIS III
Clinical trials for MUCOPOLYSACCHARIDOSIS III explained in plain language.
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One-Time gene therapy injection aims to halt devastating childhood disease
Disease control Recruiting nowThis study is testing a one-time gene therapy called UX111 for children with Sanfilippo Syndrome Type A, a rare and severe genetic disorder. The therapy involves a single intravenous injection designed to deliver a working copy of a missing gene, with the goal of slowing or stopp…
Matched conditions: MUCOPOLYSACCHARIDOSIS III
Phase: PHASE2, PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 19, 2026 14:56 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 III
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC