Optimizing DMD management in Asia: Current challenges and future directions.

Abstract

Background: Since 1970, advances in Duchenne muscular dystrophy (DMD) management have significantly extended the patients' life expectancy. However, DMD remains universally fatal.

Objective: To understand the current DMD management standards and practices in Asia and to evaluate inconsistencies, unmet needs, and future directions for DMD patient management in the region.

Methods: Clinical experts from Hong Kong, Singapore, and Taiwan convened in September 2023 to discuss DMD diagnosis, assessment, treatment, and management best practices. A pre-meeting survey was also administered to explore professional perspectives and practices and refine the discussion focus.

Results: Genetic profiling has become the pivotal diagnostic tool and has been universally adopted by experts. Steroid therapy is recognized as the cornerstone of DMD care. However, there are various opinions regarding the timing of its initiation and whether it should be continued after the loss of ambulation. Gene therapy has the potential to significantly improve outcomes in patients with DMD. However, its adoption is hampered by high costs, the potential for serious adverse events, and difficulties with patient selection. Governments and manufacturers must work together to improve its affordability. Establishing a centralized DMD registry garnered strong support from the experts as it promised to provide insights into unique DMD subtypes, standardize treatment protocols, and facilitate the conduct of clinical trials. Furthermore, the introduction of gene therapy has highlighted the need for generating long-term safety and effectiveness data in patients with DMD. Lastly, a centralized patient registry can be used to inform policy decisions.

Conclusions: DMD care in Asia is characterized by substantial heterogeneity, with financial and reimbursement issues playing a major role in management decisions. A centralized patient registry has the potential to address these concerns by providing a valuable source of information.