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Do we always need to treat patients with spinal muscular atrophy? A personal view and experience.

Caterina AgostoEleonora SalamonAntuan DivisicFrancesca BenedettiLuca GiacomelliAashni ShahGiorgio PerilongoFranca Benini
Published in: Orphanet journal of rare diseases (2021)
Our analysis further supports the findings reported in the study by Audic et al. We believe that a wider use of nusinersen in clinical practice would require a comprehensive assessment of its actual benefits weighed against the discomfort caused to patients, as well as the identification of the patients who may obtain the best benefits from this treatment.
Keyphrases
  • end stage renal disease
  • clinical practice
  • ejection fraction
  • chronic kidney disease
  • newly diagnosed
  • peritoneal dialysis
  • prognostic factors