O38: NNZ-2591, a synthetic IGF-1 metabolite analog: Phase 2 clinical trial results for children and adolescents with Pitt Hopkins syndrome
fibrosis were unchanged including patients who stopped ERT.All patients remained healthy, and no severe adverse events were reported after the initial transplant period.All patients showed polyclonal marking of peripheral blood cells and no evidence of secondary malignancies to date.
Authors
- Liza Squires (ORCID: https://orcid.org/0009-0004-6886-0020)
- Larry Glass
- Cassandra R. Newsom (ORCID: https://orcid.org/0000-0002-3706-2059)
- Nancy E. Jones
- Elliott H. Sherr
Institutions
- University of California, San Francisco (US)
- University of Alabama at Birmingham (US)
- Neuren Pharmaceuticals (New Zealand) (NZ)
Publication Details
- Journal
- Genetics in Medicine Open
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1016/j.gimo.2025.102114
- Primary Topic
- Genetics and Neurodevelopmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00