Search by Medical Condition

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z

Clinical Trials for Mucopolysaccharidosis III

• Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
• Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
• Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
• A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
• Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA
• Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
• A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
• A Treatment Extension Study of Mucopolysaccharidosis Type IIIB
• Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
• A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
• Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome
• A Treatment Study of Mucopolysaccharidosis Type IIIB
• A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
• An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
• Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
• Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
• HSCT for High Risk Inherited Inborn Errors