Sapropterin dihydrochloride for phenylketonuria

Effects of Sapropterin on Portal and Systemic Hemodynamics in Patients With Cirrhosis and Portal Hypertension: A Bicentric Double-Blind Placebo-Controlled Study

“…Sapropterin (Kuvan, Merck, London, UK), a synthetic analogue of BH4, is a novel agent approved for certain subtypes of phenylketonuria (it is considered an orphan drug) (22)(23)(24)(25). Th ough there is limited experience with its administration, it has shown a very favorable safety profi le in healthy volunteers and patients with phenylketonuria ( 26 ).…”