Turning Possibility Into Progress: The Rush F.A.S.T. Center for Translational Research Opens in Chicago
FAST and Rush University Medical Center opened a $5 million translational research center, a groundbreaking hub built to accelerate Angelman syndrome trials and train specialists.
Neuren Pharmaceuticals announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS)
Neuren Pharmaceuticals (ASX: NEU) announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). Read the full press release here. Highlights: Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed a level of improvement from baseline after 13 weeks that was statistically significant (Wilcoxon signed […]
A new publication represents a possible small-molecule approach to potentially treat the synaptic dysfunction observed in Angelman syndrome.
Peptidomimetic inhibitors targeting TrkB/PSD-95 signaling improves cognition and seizure outcomes in an Angelman Syndrome mouse model. Link to Article: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11185757/ We are excited to announce a new publication from Dr. John Marshall at Brown University made possible by FAST funding. The project started in late September 2022, aiming to modulate synaptic function with two novel […]
New FAST-TRAC grant collaboration with Dr. Alvin Huang
A FAST-funded study unveils STEP-RNP, a non-viral gene editing platform showing improved motor, cognitive function in AS mice—an early but promising step toward treating Angelman syndrome.