A Study of VRG50635 in Healthy Volunteers

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

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

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

NCT00973011 ·Status: COMPLETED ·Phase: PHASE1

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

NCT04120506 ·Status: COMPLETED ·Phase: PHASE4

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

NCT04474197 ·Status: COMPLETED ·Phase: PHASE2

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

NCT02843035 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease

NCT06858397 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

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

NCT04145037 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

NCT06308718 ·Status: TERMINATED

Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

NCT04167345 ·Status: TERMINATED ·Phase: PHASE2

A Study of Patients With Fabry Disease (US Specific)

NCT06906367 ·Status: RECRUITING

An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

NCT02107846 ·Status: COMPLETED ·Phase: PHASE2

Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

NCT01747980 ·Status: COMPLETED ·Phase: PHASE1

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)

NCT07293546 ·Status: NOT_YET_RECRUITING ·Phase: PHASE1/PHASE2

An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome

NCT07150026 ·Status: RECRUITING ·Phase: PHASE1

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

Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria

NCT00425607 ·Status: COMPLETED ·Phase: PHASE2

A Study of RG1662 in Individuals With Down Syndrome

NCT01436955 ·Status: COMPLETED ·Phase: PHASE1

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

NCT06272149 ·Status: RECRUITING ·Phase: EARLY_PHASE1

Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin in Werner Syndrome

NCT05847179 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2

A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

NCT01750957 ·Status: COMPLETED ·Phase: PHASE2

Long Term Follow-Up for Safety of AVR-RD-02

NCT06488261 ·Status: ACTIVE_NOT_RECRUITING

Extension Study of PRX-102 for up to 60 Months

NCT01981720 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Long-term Follow-Up for RGX-121

NCT04597385 ·Status: UNKNOWN