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

Aysel, 7 years

Germany

Avery Joy, 9 years

Lousiana

Ruben, 5 years

Netherlands

Defne, 7 years

Turkey

Chelsea, 4 years

Ohio

Jacob, 2 years

Connecticut

Finnley, 4 years

Wisconsin

Marie, 13 years

Germany

Marta, 9 years

Poland

Jonah, 8 years

California

Flora, 5 years

California

Conrado, 6 years

Brasil

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

Iva Milačić, PhD

Zoran Lapčević, MSc

Nicole Lytle, BA

Ružica Kravljanac, MD, PhD

Wendy Hanna-Rose, PhD

Marie Zikànovà, PhD

Olga Součková, MSc

Steven Gray, PhD

Xin Chen, MD, PhD

Ethan Perlstein, PhD

Mathura Thevandavakkam, PhD

Matt Might, PhD

Aleksandra Foksinska, MS

Nate Fehrmann

Terry Pirovolakis

Leslie Hess, MA

The content is available only in Serbian.

THANK YoU FOR YoUR CONTRIBUTIoN!

DRAGICA PASKAŠ

47,500 RSD

Natasa Marković

200 USD

Anonymous

500 USD

Your donations directly support critical research, and therapy development that will enhance the lives of these incredibly brave children and their caregivers.