Active Studies

Prospective observational drug registry developed to characterize the safety profile (including primarily potential serious hepatic risks) and to describe clinical characteristics and outcomes of patients newly treated with Opsumit in the post-marketing setting.


Purpose: The objective of this study is to evaluate the safety of long-term treatment with Kuvan.

1. PKUDOS Registry
Patients with a confirmed diagnosis of Phenylketonuria (PKU) with hyperphenylalaninemia who have either received Kuvan therapy, or currently receive Kuvan therapy, or intend to begin receiving Kuvan therapy within 90 days of entering the registry.

Study objective: Researchers are interested to find out if Vitamin D supplementation can help prevent migraine headaches in adolescents.