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The U.S. National Library of Medicine provides a database of clinical trials worldwide. Use the fields below to search the database. Expand your results and select terms or phrases and right-click to search using Google, MalaCards, MedlinePlus, PubMed, or Wikipedia.

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Clinical Trials Search Results

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Count: 169
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NCT IDTitle
NCT00445978A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
NCT03820804Nutritional Status in Phenylketonuria
NCT04368624PKU Skin Stripping
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT03505125A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT04684940Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT02468570A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
NCT03872531Lifetime Impact Study for Achondroplasia
NCT02724228A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT04055051ATHN 11: Liver Transplantation Outcomes Study
NCT06382155A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT00325962A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
NCT00838435Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
NCT01275066A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01966029BMN 110 Phase 3B in Australian Patients
NCT05734196The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
NCT01114737Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
NCT01920828Gait Analysis in MPS IVA
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT06668805A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
NCT01803412A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT02365376Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01037309Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT03150069Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
NCT07073014Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT07441876Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
NCT00484991Sapropterin Expanded Access Program
NCT00532844A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT03204370Natural History of Atypical Morquio A Disease
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT02329769Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT07126262A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
NCT03583697A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT05813314Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT01515956Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT06212947A Multicenter Multinational Observational Study of Children With Hypochondroplasia