2 results
Approved WMOCompleted
Primary ObjectivesParts 1 (Subjects with F/MF genotypes) and 2 (Optional; Subjects with the F/F genotype)* To evaluate the safety and tolerability of VX 121 in TC with TEZ/VX 561 (deuterated IVA) * To evaluate the efficacy of VX 121 in TC with TEZ/…
Approved WMOCompleted
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 (…