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Currently, caplacizumab is being developed for treatment of acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare and potentially life-threatening thrombotic microangiopathy, in which accumulation of ULvWF multimers leads to an increased…
A. What are possible causes of MP and what patients variable are asssociated with MP. What is the natural course and prognosis of MP. What is the influence of MP on the quality of life.B. What is the diagnostic value of SSEP en nerve conduction…
1. Primary Objectives• To evaluate the safety and tolerability of VX 440 monotherapy and VX 440 in dual and triple combination with VX-661 and IVA• To evaluate the efficacy of VX 440 monotherapy and VX 440 in dual and triple combination with VX 661…
The overarching goal is to optimise the treatment of KD in children/adolescents across Europe.KD-CAAP will test the hypothesis that adding immediate adjunctive corticosteroid treatment to IVIGand aspirin will reduce CAA rates in unselected KD…
Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…