Expanded Access for CC-10004

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome

NCT03882918 ·Status: TERMINATED ·Phase: PHASE3

Copper Histidinate Treatment for Menkes Disease

NCT04074512 ·Status: APPROVED_FOR_MARKETING

Safety and Efficacy of CC-10004 for Prurigo Nodularis

NCT00869089 ·Status: COMPLETED ·Phase: PHASE2

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

NCT03694353 ·Status: COMPLETED ·Phase: PHASE3

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE

NCT05030831 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

NCT02473445 ·Status: TERMINATED ·Phase: PHASE2

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

NCT03675126 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234 ·Status: COMPLETED ·Phase: PHASE3

Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

NCT01476163 ·Status: AVAILABLE

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156 ·Status: COMPLETED

Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

NCT04552691 ·Status: APPROVED_FOR_MARKETING

A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)

NCT06186492 ·Status: COMPLETED ·Phase: PHASE1

Identification of Undiagnosed Gaucher Disease

NCT01716741 ·Status: UNKNOWN ·Phase: NA

AT-007 in Adult Subjects With Classic Galactosemia (CG)

NCT05418829 ·Status: UNKNOWN ·Phase: PHASE3

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Safety and Efficacy Study of Eculizumab in Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)

NCT01410916 ·Status: COMPLETED ·Phase: PHASE2/PHASE3

Compassionate Use Study of Two Dose Levels of Thalidomide in Adults With HIV Wasting Syndrome

NCT00002157 ·Status: COMPLETED ·Phase: NA

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

NCT00875160 ·Status: TERMINATED ·Phase: PHASE1

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

NCT06488261 ·Status: ACTIVE_NOT_RECRUITING

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

NCT04783181 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

NCT00358150 ·Status: COMPLETED ·Phase: PHASE2

PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion

NCT05343715 ·Status: COMPLETED ·Phase: PHASE1

A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

NCT05139680 ·Status: COMPLETED

Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3

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

NCT00433147 ·Status: COMPLETED ·Phase: PHASE2