Cure Beta-mannosidosis
“A patient advocacy group dedicated to supporting those affected with Beta-Mannosidosis and the development of new life-saving treatments.“
What is Beta-mannosidosis?
Beta-mannosidosis (β-mannosidosis) is a rare genetic lysosomal storage disorder caused by mutations in the MANBA gene. This leads to a deficiency of the β-Mannosidase enzyme, causing toxic material buildup in cells, affecting the body and brain.
The disease varies widely in severity and can appear from infancy to adulthood. Common symptoms include intellectual disability, motor delays, speech and swallowing difficulties, vision and hearing loss, recurrent infections, seizures, scoliosis, and behavioral challenges.
There is currently no cure for this devastating disease.
Have you or a loved one been diagnosed with Beta-mannosidosis?
Register with us for more support and resources. Together, a stronger community helps us learn more about Beta-Mannosidosis and brings us closer to a cure.
Patients Affected By Beta-mannosidosis
Marco
Skylar
Oliver
Dean
Ground Breaking Research
At The Lost Enzyme Project, we are revolutionizing the approach to Beta-Mannosidosis, an ultra-rare genetic disorder. In collaboration with with leading researchers at the Kimonis Lab our focus is on developing a life-saving enzyme replacement therapy. We aim to deliver treatment that crosses the blood-brain barrier, targeting the brain and body effectively.
Where Your Support Goes
Funds raised may support research, natural history studies, therapeutic development, manufacturing, and related efforts advancing treatments for rare disease. Contributions may also help support operational costs, patient advocacy, outreach, and fundraising efforts that sustain and grow our mission.
