Pharmacists play an essential role in educating patients and caregivers about the importance of monitoring and managing the potential adverse effects associated with Elevidys, including the necessity for high-dose steroid treatments.

Patients receiving Elevidys must undergo weekly follow-ups for at least three months to track potential adverse effects such as thrombocytopenia, myocarditis, and elevated liver enzymes.

McDonald advocates for the integration of gene therapy like Elevidys into standard care for DMD, which could lead to newborn screening initiatives for earlier intervention and better health outcomes.

However, the treatment requires intensive safety monitoring, necessitating patients to commit to frequent clinic visits and laboratory tests.

Elevidys (delandistrogene moxeparvovec) marks a significant breakthrough in the treatment of Duchenne muscular dystrophy (DMD), demonstrating a remarkable increase in dystrophin production compared to current therapies.

This innovative treatment generates dystrophin levels between 30% to 50%, a substantial improvement over the 3% to 5% levels achieved by exon skipping therapies, positioning it as a transformative option for DMD patients.

Craig McDonald, MD, emphasizes that Elevidys has the potential to enhance functional abilities and maintain disease stability in patients aged four and older, with aspirations to extend its use to younger patients.

Close monitoring is crucial, as acute liver injury can occur in up to 30% of patients treated with Elevidys, which may require careful management with high-dose steroids during the treatment process.