Early research funded by the Children's Tumor Foundation has led to developments in schwannomatosis research. One award (2008) was for the development of the first mouse model for schwannomatosis with the same underlying gene mutations found in schwannomatosis patients. The second award (2011) was to support their use in a screening platform for schwannomatosis therapeutic agents.
The August issue of EMBO Molecular Medicine features an article authored by Children’s Tumor Foundation President and Chief Scientific Officer Annette Bakker, PhD, and Salvatore La Rosa, PhD, Children’s Tumor Foundation VP, Research & Development about the larger role foundations are playing within the rare disease research ecosystem. EMBO Molecular Medicine is a peer-reviewed, online open access journal dedicated to a new research discipline at the interface between clinical research and basic biology.
The NF Registry is delivering on its promise to be a valid and useful tool for both patients and researchers. This is the conclusion of an analysis published in the journal PLOS One in June 2017. Not only are thousands of patients from all over the world contributing their data to the Registry, it is being actively used, and appreciated, by researchers working on all forms of NF.
Children's Tumor Foundation Research & Development VP Salvo La Rosa was interviewed by Outsourcing-Pharma.com on how disease-focused foundations and contract research organizations (CROs) are working together to bridge the gap between drug discovery and development, a topic that will be addressed at BIO2017, the global event for biotechnology, this week.
Chronic illness in youth poses numerous challenges to the parents who care for them. Across various pediatric populations, psychological functioning in parents is compromised by the stress of having a child with special healthcare needs. This pilot study, Feasibility and Preliminary Efficacy of an Internet Support Group for Parents of a Child with Neurofibromatosis Type 1, investigated the feasibility and preliminary efficacy of an Internet Support Group (ISG) for parents of children with NF1.