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Alpha-mannosidosis is a lysosomal storage disorder caused by mutations in the MAN2B1 gene that can affect many organs and tissues throughout the body. There are three subtypes of the rare, inherited disease, and symptoms can range from mild to severe.
Management of Alpha-mannosidosis is based on symptoms and is mainly supportive, although enzyme replacement therapy has been approved to treat it in Europe. Treatments under investigation include bone marrow transplantation and gene therapy.
The latest on our research
We are jumpstarting our Alpha-mannosidosis program to accelerate research for the condition. We welcome all patients and caregivers interested in participating and spreading the word to the Alpha-mannosidosis community.