Login / Signup

Nusinersen treatment of Spinal Muscular Atrophy Type 1 - results of expanded access programme in Poland.

Sandra ModrzejewskaKatarzyna KotulskaIlona KopytaEwa GrędowskaEwa Emich-WideraKatarzyna TomaszekJustyna PaprockaDariusz ChmielewskiJacek PilchJerzy PietruszewskiAnna LemskaMarta ZawadzkaMaria Mazurkiewicz-Beldzinska
Published in: Neurologia i neurochirurgia polska (2021)
Our data indicates that nusinersen treatment might be effective in SMA type 1 patients, regardless of their age and functional status.
Keyphrases
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • peritoneal dialysis
  • prognostic factors
  • study protocol
  • machine learning
  • artificial intelligence
  • double blind