Background:
Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disorder that causes progressive muscle weakness and premature loss of function due to mutations in the DMD gene. The emergence of adeno-associated virus (AAV)–mediated microdystrophin gene therapy offers a promising disease-modifying approach. Yet, as experimental therapies move rapidly toward clinical use, early and expanded access programs raise complex ethical questions. Balancing the urgency of treatment for progressive conditions against uncertainties in safety, efficacy, and long-term outcomes remains a significant challenge for clinicians, regulators, and families.
Objective:
This review aims to explore the major ethical considerations associated with early access to gene therapy for DMD, with particular focus on informed consent, equity, and responsible clinical translation.
Methods/Approach:
A narrative review of literature published between 2018 and 2025 was conducted using PubMed and Google Scholar. Peer-reviewed articles, regulatory guidelines, and position papers from professional and patient advocacy organizations were analyzed to identify common ethical themes and recommendations related to gene therapy access.
Findings:
Four central ethical concerns emerged. First, informed consent is complicated by limited long-term safety data and uncertain efficacy. Second, justice and equity are challenged by high treatment costs, restricted eligibility, and uneven global access. Third, therapeutic misconception persists, as patients and families may equate experimental interventions with proven therapy. Finally, transparency and sustainability are critical, requiring ongoing data reporting and post-treatment monitoring to ensure accountability.
Conclusion:
Ethical governance of early gene therapy access must balance innovation with patient welfare. Standardized consent processes, equitable distribution policies, and interdisciplinary ethical oversight are essential to safeguard autonomy and justice. Proactive engagement with patient communities and regulators will be critical to ensuring responsible and transparent translation of gene therapy for muscular dystrophy.