Safety and efficacy of pegtibatinase enzyme replacement therapy in adults with classical homocystinuria in the COMPOSE phase 1/2 randomized trial
Pegtibatinase was generally well tolerated and substantially reduced tHcy levels, demonstrating potential as a treatment for HCU.
Authors
- Sharon McDermott
- Can Fıçıcıoğlu (ORCID: https://orcid.org/0000-0002-8331-9804)
- Wendy E. Smith (ORCID: https://orcid.org/0000-0003-2182-2823)
- Melissa Lah (ORCID: https://orcid.org/0000-0002-2406-8961)
- Jalé Güner
- David Kudrow
- Liz Wilkening
- Janet A. Thomas
- Jaya Ganesh
- Harvey L. Levy
- Sagar A. Vaidya
Institutions
- Boston Children's Hospital (US)
- Children's Hospital of Philadelphia (US)
- Harvard University (US)
- MaineHealth (US)
- Therapeutics Clinical Research (US)
- California Science Center (US)
- Fate Therapeutics (United States) (US)
- Indiana University School of Medicine
- Indiana University – Purdue University Indianapolis (US)
- University of Colorado Denver (US)
- Icahn School of Medicine at Mount Sinai (US)
Publication Details
- Journal
- Genetics in Medicine
- Published
- 2025-05-14
- DOI
- https://doi.org/10.1016/j.gim.2025.101456
- Primary Topic
- Folate and B Vitamins Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00