Impact of Potential Symptoms and Risks Associated with Acid Sphingomyelinase Deficiency on Patients and Caregivers: A Best-Worst Scaling Study

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 their caregivers or parents are limited. We conducted a...

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Detalles Bibliográficos
Autores principales: Mansfield, Carol, Nalysnyk, Lubomyra, Joshi, Dhaivat, Coulter, Joshua, Pulikottil-Jacob, Ruth
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2023
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10069434/
https://www.ncbi.nlm.nih.gov/pubmed/37020662
http://dx.doi.org/10.2147/PPA.S381371

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10069434/
https://www.ncbi.nlm.nih.gov/pubmed/37020662
http://dx.doi.org/10.2147/PPA.S381371

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