Expert Interview
Discussing therapies in development for Homocystinuria including Pegtibatinase from Trevere and Pegtarviliase from Aeglea
Ticker(s): TVTX, AGLEInstitution: 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 over the past 50 years and actively involved in discussions about current treatment, new ideas of pathogenesis and new treatments.
- Over 50 years 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.
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