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Table 1 Eligibility criteria

From: Evolution of mobility, pain/discomfort, self-care, and mental health in patients with alpha-mannosidosis: an international caregiver and patient survey

Eligibility criteria

Respondents had to be ≥ 18 years old to complete the survey and provide informed consent. The survey was open to:

• Patients who were ≥ 18 years old with alpha-mannosidosis

• Caregivers* responding on behalf of patients ≥ 10 years old

• Patients who had received a HSCT, those treated with ERT at the time of the study and patients who had received no specific treatment (no HSCT or ERT)

  1. * A caregiver was defined as the person who was responsible for the patient daily or at least three days per week. Caregivers with more than one child with alpha-mannosidosis were asked to complete a separate survey for each child.
  2. ERT = enzyme replacement therapy; HSCT = hematopoietic stem cell transplant
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172