Kurzfassung

A)A retrospective, non-interventional study of the data of patients diagnosed with Niemann-Pick disease type C in order to confirm ASIS as a disease progression predictive model/tool
B) 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
C) Arimoclomol prospective double-blind, randomised, placebo-controlled study in patients diagnosed with...A)  A retrospective, non-interventional study of the data of patients diagnosed with Niemann-Pick disease type C in order to confirm ASIS as a disease progression predictive model/tool
B) 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 
C) Arimoclomol prospective double-blind, randomised, placebo-controlled study in patients diagnosed with Niemann-Pick disease type C» weiterlesen» einklappen