REGENXBIO trials are currently being conducted in Canada at sites in Vancouver, Ottawa and London, Ontario. Defeat Duchenne Canada is pleased to share the latest Community Letter from REGENXBIO (see below).

REGENXBIO has finished giving RGX-202 to the last patient in its confirmatory study. This is the final study needed before the company can apply for approval. Data from both the pivotal and confirmatory parts of the AFFINITY DUCHENNE trial will be used in the application, called a Biologics License Application (BLA). REGENXBIO plans to begin this submission to the U.S. FDA in the third quarter of 2026, and is seeking approval through the FDA’s accelerated approval pathway in the second half of 2027. In May 2026, the company reported that 93% of patients in the pivotal portion showed microdystrophin (a needed protein) above the set threshold at Week 12, and that the therapy was well tolerated in 31 patients.

Please see the Community Letter and Press Release from REGENXBIO below for more details. 

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Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: