3 results
Primary Objective: To investigate the effect of treatment with nebulized rhDNase targeted to the peripheral airways compared to rhDNase targeted to the central airways on FEF75 in children with CF who are on maintenance treatment with rhDNase.…
Primary:• To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using the Time to Stand Test (…
Primary:To evaluate the feasibility and safety of intra-tumour and peri-tumour therapy with GMP hBMP4 in increasing doses in patients with progressive and/or multiple recurrent GBM, identify DLT, and to determine whether there is a maximum tolerated…