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Managing CLN2 disease: a treatable neurodegenerative condition among other treatable early childhood epilepsies.

Maria Mazurkiewicz-BeldzinskaMireia Del ToroGöknur HaliloğluHidde H HuidekoperRuzica KravljanacChris MühlhausenBrian Nauheimer AndersenIgor PrpićPasquale StrianoStėphane Auvin
Published in: Expert review of neurotherapeutics (2021)
An awareness of CLN2 disease as a potentially treatable disorder and increased understanding of the key presenting symptoms can support selection of appropriate tests and prompt diagnosis. The available enzyme replacement therapy heralds an even greater imperative for early diagnosis, and for clinicians to direct patients to appropriate diagnostic pathways.
Keyphrases
  • replacement therapy
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • smoking cessation
  • prognostic factors
  • peritoneal dialysis
  • patient reported outcomes
  • physical activity