Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)

A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

6R-BH4 Pulmonary Arterial Hypertension Study

Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders

Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions

Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors

Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction

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

A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis

Extension Study for Patients Who Have Participated in a BMN 701 Study

Sapropterin as a Treatment for Autistic Disorder

Open-Label Extension Study of Kuvan for Autism

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Rhode Island Diastolic Dysfunction – Heart Failure

Retrospective Observational Safety Effectiveness With Kuvan in hpA

Trial of Kuvan in Lesch-Nyhan Disease

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency