Kurzfassung

A phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of recombinant human acid sphingomyelinase in pediatric patients aged <18 years with acid sphingomyelinase deficiency” – mit der Substanz GZ402665 – (rekombinante humane saure Sphingomyelinase)