How FAST’s Laser Focus Benefits Translational Efforts in Neurodevelopmental Disorders More Broadly: The View from a Large Private Research Funder
Explore Dr. John Spiro’s insights on how FAST’s focus benefits neurodevelopmental disorder research, as shared at the 2023 FAST Science Summit. Learn about the Simons Foundation’s role and key breakthroughs in the field.
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.
Home-Based Video Capture to Assess Function in People Living with Angelman Syndrome
Explore the innovative use of home-based video capture to assess function in individuals with Angelman Syndrome. This method offers a sensitive, reliable outcome measure, improving clinical trials outcomes.
Hiding in Plain Sight: Dystonia in Angelman Syndrome
Explore Dr. Robert Carson’s insights on dystonia in Angelman Syndrome, its prevalence, importance of recognition, treatment options, and the role of video documentation in diagnosis.
GTP-220: A Gene Replacement Therapy Being Advanced for Angelman Syndrome
Explore the progress of GTP-220, a gene replacement therapy for Angelman Syndrome, as discussed by Dr. Jim Wilson. Learn about the challenges, disruptive potential, and future plans for this groundbreaking research.
From Benchside to Bedside: Collaboration Leads to Acceleration for Novel Delivery of CRISPR Technology
Explore the exciting advancements in CRISPR technology delivery, from benchside to bedside. Learn about the collaborative efforts, preclinical studies, and the journey of drug development.
FAST’s Roadmap to a Cure: A Year of Tough Setbacks and Huge Progress
Explore FAST’s journey towards finding a cure for Angelman Syndrome, highlighting the progress, setbacks, and future strategies. Learn about their research, clinical trials, and commitment to this cause.
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.
Development of a Drug that Strengthens Synaptic Connections for the Potential Treatment of Angelman Syndrome: The Role of BDNF
Explore the development of a drug enhancing synaptic connections for potential treatment of Angelman Syndrome. Learn about the role of BDNF and the promising results of Syn3 and D-Syn3 in restoring synaptic strength.
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.