Impact of Potential Symptoms and Risks Associated with Acid Sphingomyelinase Deficiency on Patients and Caregivers: A Best-Worst Scaling Study
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DOI: 10.2147/ppa.s381371
Abstract: Purpose Acid sphingomyelinase deficiency (ASMD) is a rare, progressive, and potentially fatal disease affecting major organs; its symptoms present heterogeneously. Data on the most bothersome symptoms for patients with ASMD types B or A/B and… read more here.
Keywords: potential symptoms; sphingomyelinase deficiency; impact potential; symptoms risks ... See more keywords