DOI: 10.1002/ccr3.73353 ISSN: 2050-0904
Clinical and Biochemical Improvement After Switching From Agalsidase Alfa to Beta in a Boy With Classic Fabry Disease: A Case Report
Nobuhiko Koga, Hiroaki Yodogawa, Takaaki Sawada, Yuichi Mushimoto, Shinichiro Nagamitsu, Kimitoshi Nakamura, Shinichi Hirose, Takahito InoueABSTRACT
We described a 12‐year‐old boy with classic Fabry disease who was diagnosed through newborn screening. At age 6.2, he started agalsidase alfa based on evidence of subclinical organ involvement. At age 7.2, acroparesthesia subsequently developed. At age 10.9, after switching to agalsidase beta due to an insufficient clinical and biochemical response, his acroparesthesia resolved and plasma Lyso‐Gb3 decreased. This report broadens the clinical understanding of children with Fabry disease.