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
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
Movement disorders affect nearly all individuals with Angelman syndrome (AS), with the most common concerns being spasticity, ataxia (as observed in the majo
The past decade has seen the development of exciting therapeutic candidates ranging from antisense oligonucleotides (ASOs) to gene replacement therapies, bot
Movement disorders affect nearly all individuals with Angelman syndrome (AS), with the most common concerns being spasticity, ataxia (as observed in the majo
Like humans with Angelman syndrome (AS), mouse models of AS exhibit seizures, impaired cognition, abnormal sleep, and motor-defects; however, the expressivit
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
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
HIC number is 2000026262 Angelman syndrome research needs your help! Volunteers are needed, individuals with Angelman syndrome (AS), aged 5-18 years of age
Additional details on the clinical trials for GTX-102 are available at the link below: https://clinicaltrials.gov/ct2/show/NCT04259281 – A Study of
FAST thanks GeneTx Biotherapeutics and Dr. Scott Stromatt for offering insight on GTX-102, the investigational antisense oligonucleotide for the treatment of