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Table 5 HCPs priorities by LSD

From: Understanding the ecosystem of patients with lysosomal storage diseases in Spain: a qualitative research with patients and health care professionals

LSD

Priority #1

Priority #2

Priority #3

Priority #4

FD

Alleviating and controlling symptoms

Early diagnosis to relatives

Strict follow-up of patient symptoms

Emotional care of the patient

GD

Avoid irreversible complications

Detect neurological deficits

Strict follow-up of patient symptoms

Keep the patient informed

PD

Early start of treatment

Preserve patient functionality

Maintain the patient’s quality of life

MPSI

Maintain the patient’s quality of life

Optimise visits to the hospital

Give a real vision of disease evolution to the patient

Performing bone marrow transplant to paediatric patient

  1. FD: Fabry disease; GD: Gaucher disease; PD: Pompe disease; MPSI: Mucopolysaccharidosis type 1