About Our Efforts
Advocating to Federal Agencies
The Food and Drug Administration (FDA) is responsible for regulating food, medicine, and devices. Your voice plays a critical role in shaping how the FDA evaluates and approves treatments for Angelman syndrome. Here's how we're making sure it's heard.
Why FDA?
- As the key agency responsible for approving therapies, we want to ensure they have a thorough understanding of Angelman syndrome.
- FDA needs to be up to date on the evolving regulatory science of AS natural history and measuring disease outcomes.
Why NIH?
- NIH is the largest funder of biomedical research in the world.
- We want to ensure NIH investment in AS is appropriately focused.
- We need to continue building strategic partnerships with the NIH.
Why Your Advocacy Matters
- Regulators make treatment approval decisions based on data and information available.
- Regulators need to understand:
- Your lived experience of AS.
- Your preferences for treatments.
- What constitutes a meaningful benefit.
- Opportunities exist to provide the information the FDA needs to make an informed decision; including direct engagement with FDA and collecting data (through surveys) to provide FDA with information on our community.