GeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome
Retrieved on:
Wednesday, May 19, 2021
Patients will then move to a maintenance phase during which they will receive GTX-102 every three months and continue to be monitored for response and safety.
Key Points:
- Patients will then move to a maintenance phase during which they will receive GTX-102 every three months and continue to be monitored for response and safety.
- In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression.
- While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently.
- GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research.