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Nusinersen in adult patients with 5q spinal muscular atrophy: A multicenter observational cohorts' study.

Juan Francisco Vázquez CostaMónica PovedanoAndrés E Nascimiento-OsorioAntonio Moreno EscribanoSolange Kapetanovic GarciaRaul DominguezJessica M ExpositoLaura GonzálezCarla MarcoJulita Medina CastilloNuria MuelasDaniel Natera de BenitoNancy Carolina Ñungo GarzónInmaculada Pitarch CastellanoTeresa SevillaDavid Hervás
Published in: European journal of neurology (2022)
Nusinersen treatment is associated with some improvements in adult SMA patients. Most severely affected patients with complex spines are probably those with the most unfavorable risk-benefit ratio.
Keyphrases
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • cross sectional
  • peritoneal dialysis
  • prognostic factors
  • clinical trial
  • young adults
  • smoking cessation
  • replacement therapy