Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

NCT01463215 ·Status: SUSPENDED ·Phase: PHASE1/PHASE2

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

NCT04836377 ·Status: TERMINATED

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197 ·Status: WITHDRAWN

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

NCT03485677 ·Status: COMPLETED ·Phase: PHASE3

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

NCT04221451 ·Status: TERMINATED ·Phase: PHASE3

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

NCT05758922 ·Status: COMPLETED ·Phase: PHASE2

Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3

NCT06523517 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2

A Long-term Follow-up Study of Gaucher Disease

NCT03190837 ·Status: RECRUITING

Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia

NCT04902781 ·Status: COMPLETED ·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 Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

NCT01422187 ·Status: COMPLETED ·Phase: PHASE3

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156 ·Status: COMPLETED

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

NCT00688597 ·Status: TERMINATED ·Phase: PHASE2

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

NCT00446550 ·Status: COMPLETED ·Phase: PHASE2

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

NCT00433147 ·Status: COMPLETED ·Phase: PHASE2

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

NCT02603562 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

NCT07281079 ·Status: RECRUITING ·Phase: PHASE3

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

NCT07054515 ·Status: RECRUITING ·Phase: PHASE3

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

NCT04002830 ·Status: COMPLETED ·Phase: PHASE4

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

NCT07082725 ·Status: RECRUITING ·Phase: PHASE3

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 Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865 ·Status: COMPLETED ·Phase: PHASE2

A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

NCT03128593 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312 ·Status: COMPLETED ·Phase: PHASE2/PHASE3