Hurler syndrome
MONDO:0011758Hurler syndrome is the most severe form of mucopolysaccharidosis type 1 (MPS1), a rare lysosomal storage disease, characterized by skeletal abnormalities, cognitive impairment, heart disease, respiratory problems, enlarged liver and spleen, characteristic facies and reduced life expectancy.
Also known as: Hurler disease, Hurler syndrome, MPS I H, MPS1H, MPSIH, mucopolysaccharidosis type 1H, mucopolysaccharidosis type IH, MPS1-H
9 clinical trials for this condition and its sub-types, 6 tagged with Hurler syndrome itself.
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Own stem cells Gene-Hacked to fight rare hurler syndrome
Disease control OngoingThis study tests a one-time gene therapy for children with Hurler syndrome, a severe genetic disorder. Doctors take the child's own blood stem cells, add a working copy of the missing gene, and put them back. The goal is to help the body produce the enzyme it lacks, potentially s…
Phase 1/2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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Gene therapy trial for rare childhood disease halted Mid-Study
Disease control PausedThis study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that damages the brain and body. The therapy delivers a working copy of the missing IDUA gene directly into the fluid around the brain. The trial aims to check if the treatment is safe a…
Phase 1/2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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New stem cell approach aims to tame rare genetic diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…
Phase 2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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Gene therapy could replace risky transplants for kids with hurler syndrome
Disease control OngoingThis Phase 3 trial tests a new gene therapy called OTL-203 against the standard stem cell transplant for children with Hurler syndrome, a rare genetic disorder. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, aiming to improve survival a…
Phase 3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC
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Tiny study tracks enzyme levels in kids with rare disease before and after transplant
Knowledge-focused OngoingThis study watches how the enzyme drug laronidase moves through the bodies of 13 children with MPS IH (a rare genetic disease) before and after they get a stem cell transplant. The goal is to learn what affects drug levels, not to test a new treatment. Children aged 0-3 who are a…
Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC
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New algorithm aims to catch ovarian damage early in young cancer survivors
Knowledge-focused CancelledThis study was designed to see if a new monitoring algorithm could detect early signs of ovarian damage in women and girls who had cancer treatment. Researchers planned to compare a group using the new algorithm with a group that received standard care. However, the study was wit…
Sponsor: University of Colorado, Denver • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:42 UTC