We raise funds for Sialidosis (Type I & Type II) medical research and give newly diagnosed families the clear, compassionate information they cannot easily find anywhere else.
Cataloguing NEU1 mutations across confirmed cases to better predict disease severity and guide genetic counseling.
Actively enrolling
Therapeutics
Enzyme Replacement Pilot
An early-phase pilot exploring neuraminidase enzyme replacement approaches in partnership with two university labs.
Year 2 of 3
Registry
Sialidosis Progression Registry Study
A longitudinal study tracking symptom progression across Type I and Type II to strengthen future clinical trial design.
Ongoing
Families who have walked this path
"When our daughter was diagnosed, we had more questions than any doctor had time to answer. The Physician Discussion Guide gave us the words we did not know how to ask for."
Maria O.
Parent of a Type II patient
"I went from feeling like the only adult with Type I Sialidosis in the world to being connected with twelve other families within a month of joining the registry."
David R.
Living with Type I Sialidosis
"As a caregiver, the financial assistance directory saved us from a stack of paperwork we did not understand. It felt like someone finally had our back."
Anjali S.
Full-time caregiver
Your gift funds the next breakthrough.
Every dollar brings researchers closer to answers and gives families the resources they need today.