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Travere Therapeutics to Present Abstracts at the Society for the Study of Inborn Errors of Metabolism Annual Symposium
SAN DIEGO, Aug. 25, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company and its collaborators will present a
About this update from Travere Therapeutics, Inc.
[{"type":"text","content":"SAN DIEGO, Aug. 25, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the Company and its collaborators will present a genetic evaluation and an analysis of cognitive function from the Company’s ongoing longitudinal natural history study of people living with classical homocystinuria (HCU), at the Society for the Study of Inborn Errors of Metabolism Annual Symposium in Freiburg, Germany, August 30 – September 2, 2022. Data exploring genomic population-based estimates of the incidence of HCU will also be presented. The Company is currently advancing a novel investigational enzyme replacement therapy, pegtibatinase, for the treatment of HCU. Authors will be present at the poster walk in Messe Freiburg on August 31, 2022, from 18:45 – 20:15 CEST. Posters will also be available electronically and displayed in the poster exhibition hall from Tuesday, August 30 until Friday, September 2. Population-based Incidence Estimates of Classical Homocystinuria Using the Genome Aggregation Database (gnomAD) Poster #: PO10-2316 A Longitudinal Study of Cognitive Function in Classical Homocystinuria Demonstrates Distinct Deficits in Inhibitory Control Poster #: PO10-2388 Insights from the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)Poster #: PO10-2629 About Classical Homocystinuria Classical homocystinuria (HCU) is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is essential for the management of methionine and cysteine in the body. Classical HCU leads to toxic levels of homocysteine that can result in life-threatening thrombotic events such as stroke and heart attacks, ophthalmologic and skeletal complications, as well as developmental delay. Current treatment options are limited to protein-restricted diet and supplemental use of vitamin B6 and betaine. About Pegtibatinase Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical homocystinuria HCU. In preclinical studies, pegtibatinase has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. Pegtibatinase is currently advancing in the ongoing Phase 1/2 COMPOSE Study to assess its safety, tolerability...