Designing The Ultimate Backstage Pass to the Brain Explore Dr. Barbara Bailus’s innovative ZIP Delivery system, a novel method to overcome the blood-brain barrier and deliver therapies directly to neurons, potentially revolutionizing neurodegenerative disorder treatments.
Economic Impact of AS: Results from Caregiver Survey Explore the economic impact of Angelman syndrome on caregivers. This article presents survey results, revealing significant costs incurred by caregivers, emphasizing the need for affordable access to treatments.
How Endpoints are Used for Drug Approval Explore the role of endpoints in drug approval, their importance in clinical trials, and how caregiver input shapes the process. Learn about the ABOM consortium’s efforts in improving treatments for Angelman syndrome.
It’s a Small World: Why FAST Is Global Explore the global reach of FAST (Foundation for Angelman Syndrome Therapeutics), its challenges, and ambitions for the coming year. Discover how FAST is making significant progress towards finding a cure for Angelman syndrome.
Patient Focused Drug Development Explore the importance of patient-focused drug development and advocacy for the rare disease community. Learn how engaging with regulators, Congress, and stakeholders can make a difference in patients’ lives.
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