Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy

Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)

NCT07185256 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

A Study of VRG50635 in Healthy Volunteers

NCT06286475 ·Status: COMPLETED ·Phase: PHASE1

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

NCT06272149 ·Status: RECRUITING ·Phase: EARLY_PHASE1

Study of STG-001 in Subjects With Stargardt Disease

NCT04489511 ·Status: COMPLETED ·Phase: PHASE2

Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease

NCT05698901 ·Status: RECRUITING

Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742 ·Status: RECRUITING

A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravitreal Injections of FCFD4514S in Patients With Geographic Atrophy

NCT00973011 ·Status: COMPLETED ·Phase: PHASE1

Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)

NCT05814952 ·Status: RECRUITING ·Phase: NA

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943 ·Status: COMPLETED ·Phase: PHASE1

Gene Therapy for Severe Crigler Najjar Syndrome

NCT03466463 ·Status: RECRUITING ·Phase: NA

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

NCT05878860 ·Status: RECRUITING ·Phase: PHASE3

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

NCT05710692 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

Fabry Disease in Cerebrovascular Disease

NCT02859363 ·Status: UNKNOWN

A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease

NCT06528080 ·Status: ACTIVE_NOT_RECRUITING ·Phase: EARLY_PHASE1

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

NCT04145037 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

NCT06926621 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE2

Extension Study of PRX-102 for up to 60 Months

NCT01981720 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)

NCT03333447 ·Status: COMPLETED

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197 ·Status: WITHDRAWN

Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)

NCT06345898 ·Status: RECRUITING ·Phase: EARLY_PHASE1

A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

NCT05791864 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Development of In Vitro Functional Assays From Primary Cells of Patients With Monogenic Diseases

NCT03763864 ·Status: ENROLLING_BY_INVITATION ·Phase: NA

Identification of Undiagnosed Gaucher Disease

NCT01716741 ·Status: UNKNOWN ·Phase: NA

To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

NCT07318909 ·Status: NOT_YET_RECRUITING ·Phase: EARLY_PHASE1