Muscular dystrophy drug pipeline offers hope for improved life expectancy

The highly diverse and innovative pipeline for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) treatment has the potential to produce therapies that will help repair the mutated gene, halt muscle degeneration, and improve patients’ life expectancy, according to new research.

Business intelligence provider GBI Research’s latest report states that 70% of all pipeline programs with disclosed molecular targets are first-in-class. This exceptional level of innovation is largely due to the high number of first-in-class products that solely target the dystrophin gene, which is the primary genetic cause of DMD and BMD.

According to Angel Wong, senior analyst for GBI Research, the pipeline offers hope of improved treatment options for a condition that has no cure available at present, as the current DMD and BMD treatment space is extremely sparse and largely symptomatic, with limited long-term benefits.