Throughout the study, no serious adverse events were reported, with only mild side effects such as nausea, vomiting, and fatigue, all of which resolved over time.

A recent clinical trial has demonstrated that gene therapy RGX-202 is both safe and effective in improving functional outcomes for boys with Duchenne muscular dystrophy (DMD).

The trial involved 12 ambulatory boys, aged between 1 and 12 years, with genetically confirmed DMD, who received a single administration of RGX-202 at two different dose levels.

Microdystrophin expression was notably detected in boys aged 4 to 7 years at the first dose level, and in younger boys at the second dose level, indicating effective delivery of the gene therapy.

The interim results of the study were presented by Dr. Aravindhan Veerapandiyan at the American Academy of Neurology Annual Meeting held in San Diego from April 5 to 9, 2025.

Further follow-up for the study is anticipated to continue for up to five years as safety monitoring remains a priority.

Significant improvements in muscle function were observed at 9 and 12 months, particularly in the North Star Ambulatory Assessment when compared to a natural history cohort.