Disease management

Managing the disease

There is currently no cure for Alpha-Mannosidosis, and treatment is often limited to addressing or controlling symptoms.1

Recommendation for symptoms management

A proactive approach is adopted to prevent any emerging complications, and a thorough clinical assessment is carried out at the time of initial diagnosis.1

Specific management options

Rationale for treatment: with this condition, all cells are without α-mannosidase activity. The rationale for HSCT in this setting is that enzyme-producing donor cells repopulate the host tissues and transfer enzyme to nearby enzyme-deficient host cells.

  1. Guffon, N. et al. Recognition of alpha-mannosidosis in paediatric and adult patients: Presentation of a diagnostic algorithm from an international working group. Molecular Genetics and Metabolism 126, 470–474 (2019).