Product information and disease descriptionPlace of product in therapyApproaches to treatmentCurrent treatment options and standard of care
Limitations of current therapies
2 evidence topics · 5 sources
Summary
Current treatment combines disease-directed therapy with rehabilitation and organ-specific supportive care. Corticosteroid adverse effects include weight gain, behavioural changes, growth suppression, bone loss, and cataracts. Exon-skipping options require mutations amenable to skipping a specified exon. The revised delandistrogene moxeparvovec indication is limited to ambulatory patients aged four years or older with a confirmed DMD mutation, and FDA identifies risks of serious liver injury and acute liver failure, including fatal outcomes. These eligibility restrictions and adverse effects limit the applicability of available therapies across the DMD population.