Educational Content
Learn about Duchenne Muscular Dystrophy, its symptoms, and treatment options.
Research Papers on Duchenne Muscular Dystrophy
Research on Duchenne Muscular Dystrophy (DMD) is progressing rapidly. Some recent studies focus on gene therapies, exon skipping, and other therapeutic approaches. Below are some key papers:
- Chimeric Cell Therapies as a Novel Approach for Duchenne Muscular Dystrophy (DMD) and Muscle Regeneration (2024) - A study discussing chimeric cell therapies as a potential treatment for Duchenne Muscular Dystrophy and muscle regeneration.
- Therapeutic Strategies in Duchenne Muscular Dystrophy: Focus on Molecular Mechanisms and Innovative Approaches (2023) - A review on recent therapeutic strategies for DMD, focusing on molecular mechanisms and novel approaches.
- Dystrophin-Associated Glycoproteins and the Pathogenesis of Duchenne Muscular Dystrophy (2003) - A foundational paper discussing the role of dystrophin-associated glycoproteins in DMD pathogenesis.
- Muscle Regeneration and the Potential for Therapy in Duchenne Muscular Dystrophy (2016) - A study focused on muscle regeneration and therapeutic options for DMD.
- Molecular Mechanisms and Potential Therapeutic Approaches in Duchenne Muscular Dystrophy (2021) - A comprehensive review on molecular mechanisms and potential therapeutic strategies for DMD.
- The Role of Inflammation in Duchenne Muscular Dystrophy and Potential Therapies (2021) - A study exploring the role of inflammation in the progression of DMD and potential therapeutic approaches.
- Genetic Modifiers of Duchenne Muscular Dystrophy: Progress and Perspectives (2019) - A study examining genetic modifiers that impact the progression and severity of DMD.
- Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management (2018) - A comprehensive guide covering early diagnosis and multidisciplinary management of DMD including neuromuscular care, rehabilitation, endocrine issues, and nutritional support.
- Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management (2018) - This paper outlines essential care strategies for managing respiratory, cardiac, bone, and orthopaedic complications in individuals with DMD.
Exon Skipping for Duchenne Muscular Dystrophy
Exon skipping is a therapeutic strategy aimed at skipping over the defective parts of the dystrophin gene, allowing the production of a shortened, but functional, form of dystrophin. This approach has shown promise in clinical trials and could provide a potential treatment option for DMD patients.
Some recent clinical trials using exon skipping technology have led to the approval of drugs like eteplirsen (Exondys 51) by the FDA, which targets exon 51 of the dystrophin gene. This therapy has been shown to improve walking ability in some patients.
For more information on exon skipping therapies, see the research paper: Exon Skipping in Duchenne Muscular Dystrophy.
Gene Therapy for Duchenne Muscular Dystrophy
Gene therapy aims to treat the root cause of DMD by either replacing the mutated dystrophin gene or by using other methods to restore the production of dystrophin. The goal is to deliver a functional copy of the dystrophin gene to muscle cells, either through viral vectors or other gene delivery mechanisms.
Currently, several gene therapy trials are underway, and there have been significant advances in the use of adeno-associated viruses (AAV) to deliver the gene therapy. Companies like Sarepta Therapeutics and Solid Biosciences are leading the development of gene therapies for DMD.
For more on the progress of gene therapies, refer to this article: Gene Therapy for Duchenne Muscular Dystrophy: Current Status and Future Directions.
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