Final results of the ASCEND trial: Continued or sustained improvements in hepatosplenomegaly, respiratory outcomes, and lipid profile after 4?years of olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency.

Melissa Wasserstein, Renata C. Gallagher, Antonio Barbato, Roberto Guigliani, Norberto Guelbert, Julia B. Hennermann, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth L. Thurberg, Nicole M. Armstrong, Shruti Srivastava, Urszula Wisniewska, Monica Kumar