P166: Safety, efficacy, and exposure-response of NNZ-2591, a synthetic analog of an IGF-1 metabolite, for Phelan-McDermid syndrome in children and adolescents*
Phelan-McDermid syndrome (PMS), a rare genetic condition associated with delayed neurodevelopment, currently has no approved treatments. The treatment effects and pharmacokinetics (PK) of NNZ-2591, a synthetic analog of cyclic glycine-proline (cGP), a metabolite of IGF-1, were evaluated in a phase 2, multisite, open-label clinical trial of children and adolescents with PMS.
Authors
- Helen Kastrissios
- Liza Squires (ORCID: https://orcid.org/0009-0004-6886-0020)
- Mark A. Milad (ORCID: https://orcid.org/0000-0002-8909-2946)
- Larry Glass
- Milad Ghomlaghi
- Nancy E. Jones
- Russell Wada
Institutions
- Australian Council for Educational Research (AU)
- Milwaukee Institute of Art & Design (US)
- IDEX Corporation (United States) (US)
Publication Details
- Journal
- Genetics in Medicine Open
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1016/j.gimo.2025.102131
- Primary Topic
- Growth Hormone and Insulin-like Growth Factors
- Type
- article
- Field-Weighted Citation Impact
- 0.00