3 results
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…
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…
This trial is, along with establishment of the safety, tolerability and pharmacokinetics and -dynamics of CDZ173 in the target population, designed to select the optimal dose to normalize the function of the PI3K protein in patients with APDS/PASLI…