Abstract
To examine the impact of the COVID-19 pandemic, we interviewed 26 patients with lysosomal storage disorders receiving enzyme replacement therapy. 20 (77 %) had significant interruption in their treatment, with an average of 8 (range 2–28) missed doses. Alternate methods of delivering uninterrupted care including home therapy were used. Vulnerable patients with chronic genetic disorders require organization for their multidisciplinary needs of care.
Keywords: Enzyme replacement therapy, Lysosomal storage disorders
Footnotes
Ethical clearance
No. IEC SGRH; EC/12/20/1784, dated January 12, 2021.
Contributors
SP, SB: conception of work, acquisition, analysis and interpretation of data, drafting and revising the work; SBM: drafting and revising the work; ICV: drafting the work and revising it critically for important intellectual content; RDP: conception and design of work, Interpretation of data, drafting and revising the work. All authors approved the final version of manuscript.
Funding
None for this study
Competing interests
None stated.
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