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Cathepsin D as biomarker in cerebrospinal fluid of nusinersen-treated patients with spinal muscular atrophy.

David C SchorlingHeike KölbelAndreas HentschelAstrid PechmannNancy MeyerBrunhilde WirthRoman Rombonull nullAlbert SickmannJanbernd KirschnerUlrike Schara-SchmidtHanns LochmüllerAndreas Roos
Published in: European journal of neurology (2022)
We identified a decline of cathepsin D levels in CSF samples of SMA patients under nusinersen treatment that was more pronounced in the group of "treatment responders" than in "nonresponders." We believe that our results indicate a suitability of cathepsin D levels as a possible biomarker in SMA also in older patients, in combination with analysis of peripheral neurofilament light chain in adolescents or alone in adult patients.
Keyphrases
  • cerebrospinal fluid
  • newly diagnosed
  • end stage renal disease
  • ejection fraction
  • physical activity
  • peritoneal dialysis
  • combination therapy