Hurler syndrome is the most severe form of mucopolysaccharidosis type 1 (MPS1), a rare lysosomal storage disease, characterized by skeletal abnormalities, cognitive impairment, heart disease, respirat...
Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 5, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 5, 2026
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Inheritance patterns describe how genetic conditions are passed from parents to children.
Age of onset indicates when symptoms typically first appear.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 5, 2026
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Common questions about Hurler syndrome
AI-Generated Content: This summary was generated using AI. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 12 companies have orphan drug designations for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
AI-curated news mentioning Hurler syndrome
Updated Feb 5, 2026
The FDA has placed clinical holds on RegenXBio's investigational gene therapies RGX-111 and another for rare neurodevelopmental disorders, including mucopolysaccharidosis type I (Hurler syndrome). This decision follows the discovery of a neoplasm in a trial participant, impacting ongoing studies.
Regenxbio's RGX-111, a gene therapy for Hurler syndrome, faces uncertainty after a cancer case was reported in its Phase 1/2 trial. This therapy aims to deliver a gene to central nervous system cells to produce the deficient enzyme, addressing the challenges of enzyme replacement therapy.
FDA has placed a clinical hold on Regenxbio's gene therapies RGX-111 and RGX-121 for severe mucopolysaccharidosis type I (MPS I) following an adverse event in a trial participant. This decision impacts the development of these therapies aimed at delivering the IDUA gene to improve patient outcomes.