The American Academy of Neurology (AAN) has recently published a new Evidence in Focus article that may be of particular interest to the TREAT-NMD network. The piece, titled “Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus,” was released on May 14, 2025, in Neurology.
This comprehensive review evaluates the current evidence regarding the efficacy and safety of delandistrogene moxeparvovec (marketed as Elevidys), a gene therapy developed for individuals with Duchenne muscular dystrophy (DMD). The article also provides key clinical considerations to support informed decision-making in practice.
In addition to the peer-reviewed article, the AAN offers several supporting materials to enhance accessibility and understanding of the findings, including:
- A clinician summary
- A patient summary
- An educational presentation
These resources are available on the AAN website alongside the full guideline.
Resources and Further Reading:
