Kurzfassung

A phase 2/3 multicenter, randomized, double-blind, placebo-controlled, repeat-dose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency

A prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression pattern

Arimoclomol prospective double‑blind,...A phase 2/3 multicenter, randomized, double-blind, placebo-controlled, repeat-dose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency

A prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression pattern

Arimoclomol prospective double‑blind, randomised, placebo-controlled study in patients diagnosed with Niemann‑Pick disease type C» weiterlesen» einklappen