A
Aicardi Goutieres Syndrome Advocacy Association
6M raised
Founded in 2018
501(c)(3)
Everything we do reflects a sense of urgency to rescue patient potential and preserve quality of life. We’re focused on accelerating research and providing timely emotional outreach and educational support alongside evolving clinical care recommendations to affected families
Therapeutics of interest
Antibodies
Cell Therapies
Therapeutics in development
Other
Members
EC
Edie Crosse
Scientific Director
BT
Betty Trevino
Marketing Director
Aicardi Goutieres Syndrome
Epidemiology
- 2 in 10000 prevalence
- 4,002 patients engaged
- 22,000 patients in the US
- 210,000 patients globally
Disease categories
Loss-of-Function
Associated genes
SAMHD1
TREX1
RNASEH2B
RNASEH2C
ADAR
IFIH1
LSM11
RNU7-1
No Conditions Yet
This organization is not associated with any conditions yet.
Research Plans
| Title & Description | Stage of Activity |
|---|---|
Drug Repurposing or Drug Screening | |
Drug Screening Research Plan This research plan is aimed at evaluating a panel of small- molecules and their effects on a disease-relevant endpoint, with the aim of identifying potential therapeutic compounds or to identify relevant and modifiable disease biology. | Preclinical |
Other | |
Untitled Research Plan | Planning |