Mucopolysaccharidosis type 2
MONDO:0010674A lysosomal storage disease leading to a massive accumulation of glycosaminoglycans and a wide variety of symptoms including distinctive coarse facial features, short stature, cardio-respiratory involvement and skeletal abnormalities. It manifests as a continuum varying from a severe to an attenuated form without neuronal involvement.
Also known as: Hunter syndrome, Hunter's syndrome, I2S deficiency, IDS deficiency, MPS 2, MPS II, MPS with skin involvement, MPS2
28 clinical trials for this condition and its sub-types, 25 tagged with Mucopolysaccharidosis type 2 itself.
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Sub-types of Mucopolysaccharidosis type 2
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Experimental cell shot aims to boost brain repair in kids with rare metabolic diseases
Disease control Recruiting nowThis early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…
Phase 1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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Lifeline for hunter syndrome patients: continued access to Brain-Targeting therapy
Disease control Expanded accessThis program offers continued treatment with idursulfase-IT (TAK-609), given directly into the spinal fluid, plus standard intravenous Elaprase, for children and adults with Hunter syndrome who have cognitive impairment. It is only open to people who completed earlier studies of …
Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC
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Milder transplant method aims to help young patients with rare blood diseases
Disease control Recruiting nowThis study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…
Phase 2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 patients with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to understand how these conditions develop and respond to treatments given before birth. Researchers will track symptoms, lab re…
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC
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Massive french study aims to unlock secrets of rare MPS diseases
Knowledge-focused Recruiting nowThis observational study will follow up to 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will collect medical data from patient records and ongoing checkups to map how the diseases progress and how current treatments affec…
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC