Discussing therapies in development for Homocystinuria including Pegtibatinase from Trevere and Pegtarviliase from Aeglea

Executed on:Sep 22, 2022 2:00 PM EDT

Call Duration:30 minutes

Expert Profile

ExpertBlinded Expert

Institution: Boston Children's HospitalSenior 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.

Interview Goal

This conversation will focus on the standard of care and landcape for Homocystinuria. We will get an expert's view on how promising the new therapies in develeopment from TVTX and AGLE are for this disorder.

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