About the Program
A therapeutic approach using a small molecule to improve synapse function by addressing BDNF signaling (Brain-Derived Neurotropic Factor), which has been shown to be abnormal in Angelman syndrome neurons. This approach improves the communication between two neurons, which is impacted in AS.
- FAST Pillar
- Pillar 4: Targeting Downstream Pathways
- Investigational Drug Name
- SYN-3
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