The ORCA (Observer-Reported Communication Ability) outcome measure
In 2018, FAST funded Dr. Bryce Reeve of Duke University to create a novel communication measurement tool as an outcome measure assessment of caregiver observ
FAST Awards Drs. Silverman (UC-Davis) and Duis (Children’s Hospital Colorado) Grant to Study Gait as an Outcome Measure for Angelman Syndrome
Movement disorders affect nearly all individuals with Angelman syndrome (AS), with the most common concerns being spasticity, ataxia (as observed in the majo
FAST Funds Dr. Albert J. Keung’s Creation of Cell Lines and Organoids of Class I Deleted Genes
The past decade has seen the development of exciting therapeutic candidates ranging from antisense oligonucleotides (ASOs) to gene replacement therapies, bot
FAST Awards Drs. Silverman (UC-Davis) and Duis (Children’s Hospital Colorado) Grant to Study Gait as an Outcome Measure for Angelman Syndrome
Movement disorders affect nearly all individuals with Angelman syndrome (AS), with the most common concerns being spasticity, ataxia (as observed in the majo
FAST Grant to Dr. Scott Dindot for Translational Research in a Pig Model of Angelman Syndrome
Like humans with Angelman syndrome (AS), mouse models of AS exhibit seizures, impaired cognition, abnormal sleep, and motor-defects; however, the expressivit
FAST Awards Grant to Dr. Yong-Hui Jiang at Yale University to Create Full Deletion Mouse Model
In 1997, Dr. Art Beaudet at Baylor College of Medicine determined that the lack of UBE3A gene expression caused Angelman syndrome (AS). Part of his finding
The ORCA (Observer-Reported Communication Ability) outcome measure
In 2018, FAST funded Dr. Bryce Reeve of Duke University to create a novel communication measurement tool as an outcome measure assessment of caregiver observ
Help create AS cell lines to test new therapeutics for each genotype
HIC number is 2000026262 Angelman syndrome research needs your help! Volunteers are needed, individuals with Angelman syndrome (AS), aged 5-18 years of age
Webinar on the KIK-AS Clinical Trials of GTX-102
Additional details on the clinical trials for GTX-102 are available at the link below: https://clinicaltrials.gov/ct2/show/NCT04259281 – A Study of
Webinar on GTX-102, an investigational antisense oligonucleotide for the treatment of Angelman syndrome
FAST thanks GeneTx Biotherapeutics and Dr. Scott Stromatt for offering insight on GTX-102, the investigational antisense oligonucleotide for the treatment of