Introduction and objective:
Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder characterized by the absence or severe deficiency of functional dystrophin, a key component of the muscle cytoskeleton and dystrophin-associated protein complex. The aim of the review is to summarize emerging therapeutic approaches currently being investigated for Duchenne muscular dystrophy, and to evaluate their potential advantages and limitations.

Review methods:
A literature search was conducted using the PubMed database. Original articles, review articles, and clinical trials published predominantly between 2020 – 2026 (95%) were included. Publications addressing the pathogenesis, genetic basis, and emerging therapeutic approaches for Duchenne muscular dystrophy were identified and analyzed following an initial screening process.

Brief description of the state of knowledge:
Duchenne muscular dystrophy is a progressive and degenerative disease that can lead to premature death. The disease primarily affects the muscular, cardiovascular, and respiratory systems. Early clinical manifestations include delayed motor development, frequent falls, and difficulty climbing stairs, which progressively contribute to the loss of independent ambulation

Summary:
Advances in the understanding of the molecular mechanisms underlying the disease have led to the development of therapies targetting the mutation itself. In recent years, considerable attention has been focused on antisense oligonucletides, CRISPR-Cas therapy, microdystrophin gene replacement, utrophin modulation, stem cell treatment, and many more. Despite these promising developments, significant challenges remain, including immunological safety concerns, efficient delivery of genetic material, and the long-term therapeutic effects.