Keynote: Dr. Allyson Berent (FAST) - How Parents Can Drive Drug Development for Rare Disease скачать в хорошем качестве

Keynote: Dr. Allyson Berent (FAST) - How Parents Can Drive Drug Development for Rare Disease

The Keynote speaker at the FOXG1 Syndrome Science Symposium and Parents Conference was the incredibly brilliant and inspiring Allyson Berent, who the FRF calls "our North star." Her presentation is titled: How Parents Can Drive Drug Development for Rare Disease: From impossible to possible for Angelman syndrome . She is the example of a rare disease parent taking charge to make a difference, and she is making a true and exciting difference. Bio: In 2014 Dr. Berent’s daughter was diagnosed with a rare non-degenerative neurogenetic disorder called Angelman syndrome. In October of 2015 she joined the Board of Directors as a Scientific Director for the Foundation for Angelman Syndrome Therapeutics (FAST), and in March of 2016 became the Chief Science Officer for the Foundation. Dr. Berent helped to spearhead the development of a pre-competitive biomarker and outcome measure consortium in order to bring patient focused outcome measures forward for human clinical trials (Angelman Syndrome Biomarker and Outcome Measure Consortium-ABOM) and now serves as the Director of this consortium. Dr. Berent Co-Founded the International Angelman Syndrome Research Council (INSYNC-AS). Through FAST, Dr. Berent collaborated with a consortium of scientists to encourage translational research opportunities, in order to help bring novel genetic therapies forward toward human clinical trials. Through this work, with the foundation, Dr. Berent co-founded GeneTx Biotherapeutics, a company singularly focused to advance an antisense oligonucleotide (ASO) therapy through IND enabling studies and a phase 1/2 clinical trial. Dr. Berent currently serves as the Chief Operating Officer of GeneTx Biotherapeutics, who partnered with Ultragenyx Pharmaceuticals in August of 2019. This program was acquired by Ultragenyx in 2022. The Phase 1/2 clinical trial started enrolling patients in February 2020 as the first intrathecally delivered ASO for Angelman syndrome, a study of safety and tolerability of GTX-102. - Help cure FOXG1 syndrome: https://foxg1research.org/donate