Early DMD data show significant impact of new gene therapy

6 March 2024

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Early data from the AFFINITY DUCHENNE trial of a new gene therapy, RGX-202, raise hopes for a breakthrough in Duchenne muscular dystrophy (DMD).

DMD is one of the most severe forms of inherited muscular dystrophies, and can lead to progressive muscle fiber degeneration and weakness.

Announcing the data, Maryland, USA-based Regenxbio (Nasdaq: RGNX) said the therapy generated “significantly increased microdystrophin expression in a 12-year-old patient."

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