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

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria