PTC-AS Gene Therapy Program Update
Get the latest update on PTC Therapeutics’ PTC-AS gene therapy program. Discover their innovative treatments for rare disorders, recent approvals, and future plans for Angelman syndrome patients.
Novel Gene Editing Approach for Long-Term Paternal Gene Activation
Explore Yale University’s novel gene editing approach for long-term paternal gene activation in Angelman Syndrome. Discover the potential of CRISPR and STA technology in gene therapy.
NNZ-2591 as a Treatment for Angelman Syndrome
Explore the potential of NNZ-2591 as a treatment for Angelman syndrome. Learn about Neuren Pharmaceuticals’ clinical trials, preclinical studies, and the drug’s neurobiological properties.
Hematopoeitic Stem Cell Gene Therapy: What is ube-cel?
Hematopoietic Stem Cell Gene Therapy: Discover the Potential of Ube-cel for Treating Angelman Syndrome. Learn about the Process and Promising Research.
Gene Therapy for Rare Genetic Neurodevelopmental Disorders: The Basics
Explore the basics of gene therapy for rare genetic neurodevelopmental disorders. Learn about the main approaches, delivery methods, and the challenges of this innovative treatment.
Fireside Chat: The FAST Commercial Philosophy
Join Alana Newhouse and Ryan Jacob as they discuss FAST’s commercial philosophy, the importance of partnerships, and the challenges in the rare disease community. Discover the balance between communication and commercialization.
FAST Roadmap to a C.U.R.E. 2.0
Join Allyson Berent at the 2022 FAST Science Summit as she discusses the roadmap to a C.U.R.E. 2.0 for Angelman Syndrome and the science behind it. Be part of the movement!
FAST Global Search & Rescue Initiative: Finding Every Individual Globally Living with Angelman Syndrome and Why it Matters
Discover the FAST Global Search & Rescue Initiative’s mission to identify every individual with Angelman syndrome worldwide. Learn about the importance, challenges, and how to get involved.
Development and Validation of a Newborn Screening Test for Angelman Syndrome
Explore the development and validation of a newborn screening test for Angelman Syndrome with Dr. Mei Baker. Learn about the integration process, follow-up care, and ethical considerations.
Crowd-Sourcing Research into Nonepileptic Myoclunus in Angelman Syndrome
Explore Dr. Carson’s research on non-epileptic myoclonus in Angelman syndrome, highlighting the importance of understanding this condition, challenges in diagnosis, and potential treatments.