Developing GTX-102, an Investigational Antisense Oligonucleotide Designed to Target & Inhibit Expression of UBE3A-AS
Time: 2:30 pm
day: Day Two
Details:
- Exploring clinical activity and evaluating the safety, tolerability, and
plasma and cerebrospinal fluid concentrations of GTX-102 in paediatric
patients - Discussing the eligibility criteria, dosage and modified trial design
chosen in this clinical trial to help inform further clinical trials - Investigate the clinical trial outcomes on patients with Angelman’s
Syndrome to help inform therapeutic development
