Qualitative interviews with patients with mucopolysaccharidosis type I (MPS I) and caregivers to evaluate the Mucopolysaccharidosis Health Assessment Questionnaire (MPS-HAQ)

Mucopolysaccharidosis I (MPS-I) is a rare autosomal recessive genetic disorder that can have physical, social, and emotional impacts. The MPS Health Assessment Questionnaire (MPS-HAQ) was developed to assess these impacts and includes 52 items across 10 domains of activities of daily living (i.e., eating/drinking, dressing, bathing, grooming, tooth brushing, toileting, mobility, walking, stairs, and caregiver assistance). It is intended for...