Work

P.33 Novel dietary management strategies for classical homocystinuria (HCU) in HARMONY/ENSEMBLE phase 3 studies of pegtibatinase, an investigational enzyme replacement therapy

Year: 2024

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2024.108399

Authors: J Thomas, T Ben-Omran, H Levy, F Maillot, S vom Dahl, S McDermott, S Slawka, C Ficicioglu

Institutions: University of Colorado Anschutz, Hamad Medical Corporation, Boston Children's Hospital, Harvard University, Université de Tours, Centre Hospitalier Universitaire de Tours, Heinrich Heine University Düsseldorf, Travere Therapeutics (United States), Children's Hospital of Philadelphia, University of Pennsylvania

Language: English

Topic: Metabolism and Genetic Disorders

Subfield: Clinical Biochemistry

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4394814044

FWCI: 0.29

Cites:

Cited by: 1

Related to: 10