At a moment of unprecedented momentum in Angelman syndrome research and therapy development, we must not let up on either government or private investment. FAST and ASF are concerned about the impact of the recent reductions-in-force (RIFs) across critical federal science agencies, including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). These agencies—and the experts who lead their work—have an essential role in advancing research and ensuring the safe, timely development of therapies for rare diseases like Angelman syndrome (AS). These actions could compromise the momentum we’ve built toward advancing meaningful potential treatments.
Angelman syndrome is a rare neurogenetic disorder that affects 1 in 15,000 individuals worldwide. Currently there are no FDA-approved treatments for AS, but we are on the verge of significant therapeutic breakthroughs—many of which have been supported through critical infrastructure, funding, and regulatory guidance provided by NIH and FDA.
The NIH is a cornerstone of rare disease innovation, supporting everything from foundational science to clinical trial readiness. Reductions in its workforce and funding will impact its ability to review and fund biomedical research. We must not take any steps that would slow progress on potential therapies or diminish the pipeline of researchers focused on rare neurodevelopmental disorders like AS.
Similarly, the FDA plays a vital role in ensuring treatments are both safe and effective. Over the past decade, the FDA has worked closely with the Angelman syndrome community to understand the unique complexities of this disorder, help build a regulatory pathway for AS-specific therapies, and enable progress in clinical trials to support drug development. We are concerned that FDA staff reductions and reorganization, and resulting loss in institutional knowledge, will undermine this progress.
FAST and ASF stand united in our commitment to drive research forward, accelerate therapeutic development, and ensure families affected by Angelman syndrome have access to the care they deserve. But we cannot do it alone. A robust NIH and high-functioning FDA are essential to our mission. We urge the Department of Health and Human Services and Congress to protect the critical infrastructure that fuels rare disease discovery and innovation. We stand ready to work in partnership with federal leaders to ensure that scientific and regulatory capabilities are strengthened—so that together, we can deliver on the promise of a better future for individuals living with Angelman syndrome.