Key result
Clinical advances and emerging molecular therapies improve management and life expectancy in Duchenne muscular dystrophy.
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Advances in clinical care and emerging molecular treatments have significantly lengthened the lifespan of patients with Duchenne muscular dystrophy, necessitating comprehensive multisystemic monitoring.
After advances in clinical care and newer efforts in therapeutic approaches, life span has lengthened in Duchenne muscular dystrophy (DMD). Starting from eary 1980s, each decade lead to a five year gain. DMD is not simply a monogenic X-linked disorder, it is a multisystemic condition. Pulmonary, cardiac, endocrine, gastrointestinal, and bone health aspects need careful monitoring along with pyschology, physiotherapy, social and family as a whole. Molecular treatments are becoming facts, which some are already at hand. Some others are expected to be available within the next two years.
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Haluk Topaloğlu (2021) conducted a review in Duchenne muscular dystrophy. Multidisciplinary care and emerging therapies was evaluated. Advances in clinical care and emerging molecular treatments, including mutation-specific strategies and gene therapy, are improving the management and life expectancy of Duchenne muscular dystrophy.
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