3 results
Approved WMOCompleted
To evaluate the safety and efficacy of PRX-102 compared to agalsidase beta in Fabry disease patients with impaired renal function.
Approved WMOCompleted
To evaluate the safety and efficacy of PRX-102 in patients with Fabry disease currently treated with agalsidase alfa
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 (…