Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency☆
Under a Creative Commons license
open access
Keywords
Olipudase alfa
Recombinant human acid sphingomyelinase
Dose escalation
Nonneuronopathic ASMD
Niemann–Pick disease type B
Cited by (0)
- ☆
Trial registration: Clintrials.gov trial registration # NCT01722526.
Copyright © 2015 Published by Elsevier Inc.