Expert Interview
Discussing PTC Therapeutics' FDA Approval of Sephience (sepiapterin) for Patients With Phenylketonuria (PKU)
Ticker(s): PTCTInstitution: Boston Children's Hospital
- Senior Physician in Medicine and Genetics at Boston Children's Hospital & Professor of Pediatrics at Harvard medical School.
- Diagnosed, treated and followed approximately 40 patients with galactosemia
- Specializes in the research, diagnosis and treatment of inborn errors of metabolism. Investigator for the national study of the natural history and treatment of urea cycle disorders, a study examining brain imaging in PKU, and a study to determine the feasibility of next generation sequencing in newborn screening.
Describe your background and practice setting
Added By: ben_adminHow many patients with PKU do you currently manage?
Added By: ben_adminCan you describe the standard of care for PKU?
Added By: ben_adminWhere will Sepiapterin fit into your treatment algorithm for PKU?
Added By: ben_adminRank your excitement for Sepiapterin on a scale of 1-10
Added By: ben_adminAre You Interested In These Questions?
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