E-Book

E-Book 3rd Congress

  • the new treatments of Duchenne muscular dystrophy (DMD)
  • Ghazale Borvaye,1,* dr Issa Layali,2 Hediye geravandi,3 zaynab kanani nejad,4 Armina aghazade,5
    1. department of microbiology, Islamic Azad university, medical branch, Tehran,Iran
    2. Department of Biochemistry and Biophysics, Faculty of Advanced Sciences and Technology, Tehran Medical Sciences Branch, Islamic Azad University, Tehran, Iran
    3. Department of Medicine,Ahvaz Jondishapur University of Medical Sciences, Khuzestan, Iran
    4. department of Laboratory Science, Islamic azad university, borujerd, Iran
    5. department of microbiology, Islamic Azad university, medical branch, Tehran,Iran


  • Introduction: Genetic disease Duchenne muscular dystrophy is one of the severe genetic diseases that occurs in children, and it happens due to deletion or disruption in the protein dystrophin, which is encoded by the gene located on the X chromosome. This disease mostly occurs in boys, and out of every 3500 male children, one is usually affected by this disease. Until now, there hasn't been a completely effective treatment method that has received FDA approval. However, new therapeutic approaches are being investigated and tested, which can potentially create a significant impact and slow down the progression of the disease. These approaches include the following cases. Genetic approaches, such as gene replacement therapies and micro-dystrophin gene therapy, along with steroid therapies like prednisone and deflazacort, anti-inflammatory drugs, including utrophin modulators, gene editing technologies like CRISPR-Cas9, read-through of premature stop codons using Ataluren, Eteplirsen, which works to promote dystrophin production by specific skipping of exon 51 in ineffective gene variants, are all potential treatments for Duchenne muscular dystrophy (DMD). In this article, we will try to discuss the mentioned innovative therapeutic methods but It's important to note that while these treatments hold promise, many are still in the experimental stage or undergoing clinical trials.
  • Methods: We use a wide variety of information that has been collected by researchers through patient samples, diverse methods, and tools. Our goal is to examine innovative therapeutic solutions for Duchenne muscular dystrophy. In addition, we evaluate the efficacy and safety of therapeutic strategies in order to improve the treatment of this disease, such as genetic therapies, steroid treatments, and so on. The integration of these materials and methods helps in designing innovative treatment options for Duchenne muscular dystrophy.
  • Results: All the mentioned methods in the experimental stages are inconclusive and do not definitively lead to complete disease improvement. Since Duchenne muscular dystrophy patients have a low life expectancy (about twenty to thirty years), helping reduce disease progression through new therapeutic methods can contribute to increasing lifespan and reducing disease severity.
  • Conclusion: Based on promising new research, we hope that by providing new treatment solutions, we can assist in improving the course of Duchenne muscular dystrophy. Generally, considering muscular, cardiac, and respiratory diseases that patients suffer from, the best therapeutic approach is prescribing various complementary medications to treat associated diseases, using assistive devices for facilitating movement, and utilizing physiotherapy and genetic therapies. It is hopeful that we can slow down the disease progression and witness improvement in the course of Duchenne muscular dystrophy patients.
  • Keywords: dystrophy, treatment, Duchenne,