A fight the world has never seen before
Join Erik in beating ASNSD
Erik has been diagnosed with asparagine synthetase deficiency (ASNSD). This nano-rare disease has no treatment and very poor prognosis. But we are here to change that. With your help we can beat the disease and save Erik's life.
Follow his story on our social media channels:
Help Us Develop the Cure
ASNSD Research Association is a parent-led global organization that drives research to develop successful therapeutic approaches for all patients with asparagine synthetase deficiency.
THERE IS HOPE for patients impacted by this rare genetic disease and we are here to bring leading international experts together and support parent advocacy.
Research to get to cure
01 Project AAV
AAV gene therapy approach that includes proof of concept and translational stages
02 Project ASO
Development of splice switching antisense oligonucleotides (ASOs)
as N-of-1 therapy
03 Foundational research
Better understand the underlying biology and mechanisms of ASNSD to foster better disease management
04 Drug repurposing
Exploring metabolic supplements, pathway modulators and neuroprotective agents
Upcoming Events
Join us for the following events
Family day to support ErikSun 14 DecTrélex