Study of rhASB in Patients With Mucopolysaccharidosis VI

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

NCT02618512 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

AVTX-801 D-galactose Supplementation in SLC35A2-CDG

NCT05402384 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144 ·Status: COMPLETED ·Phase: PHASE3

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

NCT00214773 ·Status: COMPLETED

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

NCT04201405 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Gene Therapy in Patients With Mucopolysaccharidosis Disease

NCT03173521 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

NCT02293408 ·Status: TERMINATED

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

NCT03612869 ·Status: UNKNOWN ·Phase: PHASE2/PHASE3

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

NCT05422482 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769 ·Status: RECRUITING ·Phase: PHASE1

Long-term Follow-Up for RGX-121

NCT04597385 ·Status: UNKNOWN

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

NCT02418455 ·Status: COMPLETED ·Phase: PHASE2

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCT02702115 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation

NCT06556394 ·Status: RECRUITING ·Phase: PHASE1

Repurposing 5-Azacytidine for the Treatment of Muscle Contractures in Children With Cerebral Palsy

NCT06377085 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE1

BMN 110 Phase 3B in Australian Patients

NCT01966029 ·Status: COMPLETED ·Phase: PHASE3

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

NCT00912925 ·Status: COMPLETED ·Phase: PHASE3

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

NCT02293382 ·Status: COMPLETED

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

NCT02350816 ·Status: TERMINATED ·Phase: PHASE2

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07082543 ·Status: RECRUITING ·Phase: PHASE3

A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy

NCT03570931 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2/PHASE3