Work

Pegtibatinase, an Investigational Enzyme Replacement Therapy for the Treatment of Classical Homocystinuria: Initial Results From the Phase 1/2 Compose Study

Year: 2022

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2022.01.060

Authors: Harvey L. Levy, Janet Thomas, Can Ficicioglu, Melissa Lah, Jaya Ganesh, Marcia Sellos-Moura, Erez M. Bublil, Elizabeth Wilkening, Jalé Güner, Ying Chen, Feriandas Greblikas, Sagar A. Vaidya

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

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: W4220717683

FWCI: 0.174

Cites:

Cited by: 1

Related to: 10