A recent analysis of 9000 publications reveals growing interest in precision medicine and gene therapies for Duchenne muscular dystrophy (DMD) research. Since 2004, the number of DMD-related studies has increased steadily, with a surge in publications beginning in 2014. The US leads global publication output, followed by France’s Institut National de la Santé et de la Recherche Médicale.
Researchers focus on advanced therapeutic strategies, such as gene editing, exon-skipping therapies, and systemic delivery methods for mRNA. Emerging technologies like CRISPR-Cas9 enable targeted genetic mutation correction within the DMD gene. Precision medicine aims to tailor treatments based on individual patient factors, but challenges remain due to genetic complexity and delivery system optimization.
Key hotspots in DMD research include CRISPR, eteplirsen, mitochondria, MRI, cognition, and structural variants. Studies highlight the importance of multidisciplinary care and integrating emerging therapies to improve patient outcomes. The analysis emphasizes the need for lifelong care plans that include respiratory and cardiac management, glucocorticoid therapy, and orthopedic interventions.
The goal remains consistent: enhancing muscle function, delaying disease progression, and improving quality of life. This shift towards a multidisciplinary, patient-centered approach combines cutting-edge innovations with holistic care practices, paving the way for precision medicine to transform DMD treatment.
Source: https://www.ajmc.com/view/study-highlights-trends-emerging-innovations-in-dmd-research