Adults With Acid Sphingomyelinase Deficiency Have Sustained Improvements in Clinical Outcomes With up to 5 Years of Olipudase Alfa Enzyme Replacement Therapy: ASCEND Trial Final Results. uri icon

authors

  • Wasserstein, Melissa P
  • Lidove, Olivier
  • Mabe, Paulina
  • Mengel, Eugen
  • Scarpa, Maurizio
  • Senates, Ebubekir
  • Tchan, Michel
  • Thurberg, Beth L
  • Villarrubia, Jesus
  • Armstrong, Nicole M
  • Bhushan, Shashi
  • Hollak, Carla E
  • Kim, Yong
  • Manish Kumar, MD
  • Barbato, Antonio
  • Gallagher, Renata C
  • Giugliani, Roberto
  • Guelbert, Norberto Bernardo
  • Hennermann, Julia B
  • Ikezoe, Takayuki
  • Lachmann, Robin

publication date

  • May 2026