OUR MISSION
Our mission is to offer hope and provide a brighter future to children affected with adenylosuccinate lyase (ADSL) deficiency. We are committed to advancing research and developing effective treatment that will alleviate their symptoms and improve their quality of life. Our ultimate goal is to enable gene therapy, the only way to permanently improve their health. Your support through donations is essential in helping us achieve this life-changing mission. Together, we can make a difference.
RESEARCH
Two research programs are underway with the shared goal of developing specific and effective therapy for ADSL deficiency. One project focuses on drug repurposing, testing the efficacy of approved, existing drugs in treating animal models of ADSL deficiency. The other, more complex and longer-term project, is gene therapy replacement. This project aims to deliver a functional and therapeutic ADSL gene into the central nervous system (CNS) of patients with ADSL deficiency, with the goal of permanently improving their health.
NEWS
Minja's mother is discussing ADSL deficiency and the journey to develop therapy that proves to be effective for this rare disease.
[YoUTUBE video]
SOURCE: Prva TV
DATE: 18.1.2024.
LET’S Do THIS ToGETHER FoR THeIR OPPoRTUNITY TO LIvE BETTER AND HAPPIER LIVeS
BASIC FACTS ABoUT ADSL DeFICIENCY YoU NEED TO KNoW:
ADSL (adenylosuccinate lyase) deficiency is a rare genetic disorder affecting purine metabolism and causing various clinical symptoms.
Common symptoms include seizures, delayed psychomotor development, muscle ataxia, autistic features, hypotonia...
There is no approved therapy for ADSLd.
Advancing research and developing effective therapy are vital for enhancing the lives of children with ADSLd.
Gene therapy holds a promise for treating this severe neurometabolic disease.
ADSLd TEAM
The content is available only in Serbian.
THANK YoU FOR YoUR CONTRIBUTIoN!
Your donations directly support critical research, and therapy development that will enhance the lives of these incredibly brave children and their caregivers.
