alpha-dna

DISEASE INFORMATION

alpha-cartella

Symptoms & diagnosis

Disease Management

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.2
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Specific management options

  • Hematopoietic stem cell transplantation (HSCT)34 HSCT can be a treatment option for some patients, however the riskbenefit profile is more favourable in younger patients, therefore ensuring an early diagnosis is critical for this to be a real option for patients. The rationale is that enzyme-producing donor cells repopulate the host tissue and transfer enzyme to nearby enzyme-deficient host cells.
  • Enzyme Replacement Therapy (ERT)56 is a therapeutic alternative in a number of lysosomal storage diseases,7 which replaces the missing enzyme, modifying the underlying cause of the disease. ERT is given via repeated intravenous infusion, to reduce the symptoms and clinical manifestations associated with the disease.
    An ERT is approved for use in the European Union.8
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GENERAL MANAGEMENT

SPECIFIC MANAGEMENT

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