Clinical Trial: Trial of Kuvan in Lesch-Nyhan Disease

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional




Official Title: Trial of Kuvan™ (Sapropterin) Treatment in Patients With Lesch Nyhan Disease

Brief Summary: To assess the possibility that treatment with Kuvan (a form of tetrahydrobiopterin) will lessen the abnormal behavior and/or neurology commonly found in Lesch-Nyhan disease (LND); to assess biochemical changes as measured in blood and urine.