Expert Interview
A Second Look: Discussing PTC Therapeutics' FDA Approval of Sephience (sepiapterin) for Patients With Phenylketonuria (PKU)
Ticker(s): PTCTInstitution: Childrens National Hospital
Director of the Inherited Metabolic Disorders Program at Children’s National Hospital and Associate Professor of Pediatrics at The George Washington University.
Manages 18 patients with either PKU or Hyperphenylalaninemia (H-PHE), and 3 with PTPS deficiency.
NIH- and PCORI-funded investigator; site PI for the Urea Cycle Disorders Consortium and widely published and internationally recognized speaker on metabolic disorders.
Describe your background and practice setting
Added By: nick_adminHow many patients with PKU do you currently manage?
Added By: nick_adminCan you describe the standard of care for PKU?
Added By: nick_adminWhere will Sepiapterin fit into your treatment algorithm for PKU?
Added By: nick_adminRank your excitement for Sepiapterin on a scale of 1-10
Added By: nick_adminAre You Interested In These Questions?
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