Testing effectiveness (Phase 2)Study completedNCT04817111What this trial is testingNAD+ Precursor Supplementation in Friedreich's AtaxiaWho this might be right forFriedreich Ataxia Metro International Biotech, LLC 7
Testing effectiveness (Phase 2)Study completedNCT01885936What this trial is testingSafety and Efficacy of Albuterol in Individuals With Late-onset Pompe DiseaseWho this might be right forPompe Disease Duke University 16
Not applicableStudy completedNCT02403687What this trial is testingProspective Analgesic Compound Efficacy (PACE) StudyWho this might be right forArthritisTendonitisGout+5 more Express Specialty Pharmacy 300
Testing effectiveness (Phase 2)Study completedNCT02004704What this trial is testingA Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencyWho this might be right forSphingomyelin Lipidosis Genzyme, a Sanofi Company 25
Not applicableStudy completedNCT03820804What this trial is testingNutritional Status in PhenylketonuriaWho this might be right forPhenylketonurias Universidade do Porto 94
Not applicableStudy completedNCT01610011What this trial is testingAcute Glycine Pharmacodynamic StudyWho this might be right forSchizophreniaPsychotic Disorders Mclean Hospital 21
Not applicableLooking for participantsNCT05128149What this trial is testingMetabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?Who this might be right forPhenylketonurias University Children's Hospital, Zurich 200
Not applicableUnknownNCT03840928What this trial is testingPatientSpot Formerly Known as ArthritisPowerWho this might be right forRheumatoid ArthritisAnkylosing SpondylitisFibromyalgia+61 more Global Healthy Living Foundation 40,000
Early research (Phase 1)Study completedNCT02240407What this trial is testingRe-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe DiseaseWho this might be right forPompe Disease University of Florida 2
Not applicableStudy completedNCT02354664What this trial is testingDynamic Respiratory Muscle Function in Late-Onset Pompe DiseaseWho this might be right forPompe Disease University of Florida 14
Not applicableActive Not RecruitingNCT04612764What this trial is testingLiver Disease in Urea Cycle DisordersWho this might be right forUrea Cycle DisorderOrnithine Transcarbamylase DeficiencyCitrullinemia 1+5 more Baylor College of Medicine 62
Testing effectiveness (Phase 2)Study completedNCT02489292What this trial is testingStudy to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)Who this might be right forUrea Cycle Disorders Cellaion SA 5
Post-approval studies (Phase 4)Study completedNCT02405598What this trial is testingEvaluation of Salbutamol as an Adjuvant Therapy for Pompe DiseaseWho this might be right forPompe Disease National Taiwan University Hospital 14
Not applicableWithdrawnNCT05041114What this trial is testingSWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With ParalysisWho this might be right forNeurologic DisorderParalysisParalysis; Stroke+7 more Synchron Medical, Inc.
Not applicableStudy completedNCT02801539What this trial is testingRespiratory Muscle Training in L-Onset Pompe Disease (LOPD)Who this might be right forGlycogen Storage Disease Type II Duke University 22
Not applicableEnrolling By InvitationNCT07635030What this trial is testingGABA and GSH in FRDAWho this might be right forFriedreich's AtaxiaFRDA Children's Hospital of Philadelphia 60
Large-scale testing (Phase 3)WithdrawnNCT05815004What this trial is testingAn Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3Who this might be right forGaucher Disease, Type 3 AVROBIO
Not applicableLooking for participantsNCT05698901What this trial is testingBiomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry DiseaseWho this might be right forFabry Disease Mackay Memorial Hospital 150
Testing effectiveness (Phase 2)Study completedNCT02660112What this trial is testing(+) Epicatechin to Treat Friedreich's AtaxiaWho this might be right forFriedreich's Ataxia Ralitza Gavrilova 10
Testing effectiveness (Phase 2)Study completedNCT00763932What this trial is testingExtension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) StudiesWho this might be right forPompe Disease Infantile-OnsetGlycogen Storage Disease Type II Genzyme, a Sanofi Company 7