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.
Clinical Trial Basics: What Parents Need to Know About Trial Participation
Explore the basics of clinical trials for parents considering participation for their children with Angelman Syndrome. Understand the phases, control groups, consent process, and how to make an informed decision.
Breaking the Mold: How Patient Groups like FAST are Reshaping Drug Development in Rare Disease
Explore how patient groups like FAST are revolutionizing drug development in rare diseases. Discover the importance of prevalence, progress, pathways, and partnerships in this process.