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.
DAY 2 Question and Answer Panel | 2023 FAST Summit on Angelman Syndrome
Get insights from the 2023 FAST Science Summit’s Q&A panel on Angelman Syndrome. Learn about Phase 3 trials, combination therapies, gene therapy future, and more from experts in the field.
DAY 1 Question and Answer Panel | 2023 FAST Summit on Angelman Syndrome
Get key insights from the 2023 FAST Summit’s Q&A panel on Angelman Syndrome. Learn about the latest research, the importance of community participation, and the promising future of treatment.
Clinical Development Update: NNZ-2591 as a Treatment for Angelman Syndrome
Explore the latest updates on NNZ-2591, a promising treatment for Angelman syndrome by Neuren Pharmaceuticals. Learn about its clinical development, study progress, and future plans.