Treatment of Duchenne Muscular Dystrophy with Confirmed DMD Gene Mutation in Ambulatory Patients Aged 4 and Older

This protocol addresses a specific, well-characterised sub-population of Duchenne muscular dystrophy: ambulatory patients aged 4 years or older with a confirmed mutation in the DMD gene who meet defined molecular eligibility criteria.

Patient Population

Patients eligible for this protocol must meet all of the following criteria:

Age 4 years or older
Confirmed mutation in the DMD gene
Ambulatory

Not eligible if any of the following apply:

Exon 8 or exon 9 deletion in the DMD gene
Pre-existing antibodies to adeno-associated virus

Treatment Approach

For patients who meet the above criteria, management in this setting involves an AAV-based gene therapy given as a single administration — the complete regimen, confirmatory steps, and clinical decision pathway are available in the full protocol.

Instant Access to Structured Evidence-Based Regimens

References

Elevidys is a single-dose adeno-associated virus (AAV)-based gene therapy indicated for the treatment of patients 4 years or older who have a confirmed mutation in the DMD gene and are ambulatory.

People with certain mutations (exons 8 or 9 deletions) or who have antibodies to the adeno-associated virus are ineligible to receive Elevidys.

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