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

A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease

A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease

Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors

Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors

Study of Kuvan Treatment in Adults With GTPCH Deficiency

The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease