The USFDA has approved a new therapy- casimersen for a rare genetic disorder, Duchenne muscular disorder (DMD) . DMD is characterized by muscular dystrophy caused by DMD gene mutations resulting in the absence of dystrophin, a muscle fiber protein. Casimersen injection is the first approved therapy for the treatment of patients having a mutation in the DMD gene amenable to exon 45 skipping . The approval was based on clinical trial results obtained from a double-blind, placebo-controlled study involving 43 patients with DMD.
The study population included male patients in the age group 7 to 20 years and were randomized in a 2:1 ratio to receive either intravenous casimersen injection (30 mg/kg) or placebo. Over the study period of 48 weeks, the patients in the casimersen treated group showed a significant increase in the dystrophin protein level in the body than the placebo group. The…
