FAST and ASF Advocate for Crucial Legislation
ASF and FAST sign a joint letter supporting key legislation—the PRV Program, MVP Act, and Accelerating Kids Access to Care Act—to speed treatment access for the Angelman community.
FAST and ASF Announce their Partnership with the Institute for Gene Therapies
ASF and FAST partner with the Institute for Gene Therapies to push for policy changes that ensure patients can access cutting-edge gene therapies for Angelman syndrome.
RSVP for the January 2025 EL-PFDD Meeting
FAST and ASF will co-host a virtual Externally Led-PFDD Meeting on January 29, 2025, giving the Angelman syndrome community a direct voice with the FDA on therapy development.
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.
The National Institutes of Health awarded the first phase of a roughly $40M grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases
In October 2023, the National Institutes of Health awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome. The project team named under […]