Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy
Effectiveness of elapegademase was maintained up to 4 years of use and with no new safety concerns.
Authors
- Michael S. Hershfield (ORCID: https://orcid.org/0000-0003-3589-8554)
- Leo Lin
- Michael D. Keller (ORCID: https://orcid.org/0000-0001-8323-3085)
- Anna Rozova
- Jolán E. Walter (ORCID: https://orcid.org/0000-0002-6192-0703)
- Tracy Fausnight (ORCID: https://orcid.org/0000-0002-0251-2486)
- Luke A. Wall (ORCID: https://orcid.org/0000-0001-9125-8530)
- Teresa K. Tarrant (ORCID: https://orcid.org/0000-0003-4067-5363)
- Manish J. Butte (ORCID: https://orcid.org/0000-0002-4490-5595)
- Noemi Toiber Temin
- Susan Laubach (ORCID: https://orcid.org/0000-0002-7470-9616)
- Jay R. Lieberman (ORCID: https://orcid.org/0000-0002-1374-8929)
- Jeffrey J. Bednarski (ORCID: https://orcid.org/0000-0001-6902-0876)
- Hey Chong (ORCID: https://orcid.org/0000-0002-6938-9267)
- Heather K. Lehman (ORCID: https://orcid.org/0000-0002-7553-9751)
- Bob Geng (ORCID: https://orcid.org/0000-0002-4618-4445)
- Talal Mousallem (ORCID: https://orcid.org/0000-0002-3692-8930)
- Morna J. Dorsey (ORCID: https://orcid.org/0000-0002-9432-3542)
- Tamara C. Pozos (ORCID: https://orcid.org/0000-0003-0763-8763)
- Caroline Fradette
Institutions
- Louisiana State University (US)
- Children's National (US)
- Johns Hopkins University (US)
- University of Tennessee Health Science Center (US)
- University of California, Los Angeles (US)
- University of Pittsburgh (US)
- Duke University (US)
- Washington University in St. Louis (US)
- University of South Florida (US)
- Minnesota West Community & Technical College (US)
- University of California San Diego (US)
- Johns Hopkins All Children's Hospital (US)
- University of California San Francisco Medical Center (US)
- Children's Minnesota (US)
- Centre for Global Health Research (CA)
- Jacobs Institute (US)
- University at Buffalo, State University of New York (US)
- Louisiana State University Health Sciences Center New Orleans (US)
- Penn State Milton S. Hershey Medical Center (US)
Publication Details
- Journal
- Journal of Clinical Immunology
- Published
- 2025-03-27
- DOI
- https://doi.org/10.1007/s10875-025-01873-3
- Citations
- 2
- Primary Topic
- Immunodeficiency and Autoimmune Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.59
Funders
- Chiesi USA