Work

P.10 Latest results from the COMPOSE® Phase 1/2 trial of pegtibatinase, a novel investigational enzyme replacement therapy for classical homocystinuria (HCU)

Year: 2024

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2024.108376

Authors: J Ganesh, J Thomas, W Smith, M Lah, D Kudrow, J Güner, S McDermott, S Vaidya, L Wilkening, H Levy, C Ficicioglu

Institutions: Icahn School of Medicine at Mount Sinai, University of Colorado Anschutz, Maine Medical Center, Indiana University School of Medicine, Travere Therapeutics (United States), Boston Children's Hospital, Harvard University, 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: W4394823394

FWCI:

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