Mucopolysaccharidosis I
Pipeline by Development Stage
Competitive Landscape
4 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (13)
Total enrollment: 161 patients across 13 trials
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Clinical Trial of Growth Hormone in MPS I, II, and VI
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
A Study of JR-171 in Patients With Mucopolysaccharidosis I
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
Immune Tolerance Study With Aldurazyme® (Laronidase)
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Phase Legend
Key Insights
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.