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At the present, the best treatment presently available is symptomatic management.
There is no complete cure for Duchenne muscular dystrophy, but coordinated, multidisciplinary care meaningfully extends both survival and quality of life, and the treatment landscape has changed substantially over the past decade.
• Corticosteroids, typically prednisone or deflazacort, are the cornerstone of treatment once a plateau or decline in motor skills is noticed, usually between ages four and eight. They measurably slow the loss of muscle strength and function, delay loss of independent walking, and are often continued after walking is lost to help preserve arm strength and slow the progression of scoliosis and breathing decline, though they carry real trade-offs including weight gain, slowed growth, and increased fracture risk that require ongoing monitoring.
• Exon-skipping therapies are a newer class of genotype-specific treatments that use synthetic molecules to restore the disrupted reading frame in certain genetic changes, allowing the body to produce a shortened but partially functional dystrophin protein. Several have received United States regulatory approval, each applicable only to individuals whose specific genetic change is amenable to skipping a particular exon, together covering roughly thirty percent of individuals with DMD.
• Cardiac medications, including ACE inhibitors, angiotensin receptor blockers, and beta blockers, are used, often starting before symptoms appear, to protect and support heart function, since heart involvement affects nearly everyone with DMD by adulthood.
• Gene transfer therapy, using viral vectors to deliver a shortened, functional version of the dystrophin gene, has moved from clinical trials toward wider use in recent years and is applicable regardless of a person’s specific genetic change, though it cannot restore full-length dystrophin and its long-term durability is still being studied.
• Orthopaedic and rehabilitative care includes physical therapy to maintain mobility and prevent joint contractures, bracing or spinal surgery for scoliosis, and attention to bone health, including vitamin D and calcium optimization, given the fracture risk associated with reduced mobility and corticosteroid use.
• Respiratory support, including assisted cough techniques and, over time, non-invasive or invasive ventilation, becomes increasingly important as breathing muscles weaken, particularly after independent walking is lost.
Additional supportive measures include nutritional assessment, annual influenza and pneumococcal vaccination, and specific anesthesia precautions, since individuals with dystrophinopathies can react severely to certain anesthetic agents. There are treatment trials available in India, you can refer [https://genetidoc.com/rarediseaseforum/forums/forum/genetic-trials-registry/indian-trials/duchenne-muscular-dystrophy-dmd/] for more information on them
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