A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease

Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy

NCT07119775 ·Status: AVAILABLE

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

NCT01889862 ·Status: COMPLETED ·Phase: PHASE3

A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

NCT00884949 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

BMN 110 US Expanded Access Program

NCT01858103 ·Status: APPROVED_FOR_MARKETING

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01515956 ·Status: COMPLETED ·Phase: PHASE2

Cerliponase Alfa Observational Study in the US

NCT04476862 ·Status: ACTIVE_NOT_RECRUITING

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

NCT01609062 ·Status: TERMINATED ·Phase: PHASE2

New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis

NCT02583152 ·Status: UNKNOWN

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01242111 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

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

NCT03570931 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2/PHASE3

Suitability of Nitisinone in Alkaptonuria 2

NCT01916382 ·Status: UNKNOWN ·Phase: PHASE3

24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG

NCT05549219 ·Status: COMPLETED ·Phase: PHASE2

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

Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients

NCT06657859 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE2

Gene Therapy for Children With CLN3 Batten Disease

NCT03770572 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

NCT02968953 ·Status: NO_LONGER_AVAILABLE

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

NCT06706388 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease

NCT00060515 ·Status: TERMINATED ·Phase: PHASE1

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

NCT05174039 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01275066 ·Status: COMPLETED ·Phase: PHASE3

Safety and Efficacy of CC-10004 for Prurigo Nodularis

NCT00869089 ·Status: COMPLETED ·Phase: PHASE2

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

NCT03231878 ·Status: COMPLETED ·Phase: PHASE2/PHASE3

A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria

NCT06147856 ·Status: WITHDRAWN ·Phase: PHASE1/PHASE2

BMN 110 Phase 3B in Australian Patients

NCT01966029 ·Status: COMPLETED ·Phase: PHASE3

Study of Acamprosate in Fragile x Syndrome

NCT01911455 ·Status: COMPLETED ·Phase: PHASE1