The ASNSD Research Association is a parent-led global nonprofit organization focused on advancing research for people with asparagine synthetase deficiency (ASNSD), a rare genetic disease with no established treatment and a poor prognosis. Its mission is to develop successful therapeutic approaches for all affected patients by bringing leading international experts together and supporting parent advocacy.

The association organizes its work across four research priorities. Project AAV pursues a gene therapy approach through proof-of-concept and translational stages. Project ASO develops splice-switching antisense oligonucleotides as potential individualized N-of-1 therapy. Foundational research investigates ASNSD’s underlying biology and mechanisms to inform disease management, while drug repurposing explores metabolic supplements, pathway modulators, and neuroprotective agents. The organization also shares patient stories and information for researchers and families, and raises support through donations and events.

Registered as a public interest nonprofit in Canton Vaud, Switzerland, the association states that 100% of donations support medical research and that it has no paid employees.