Abstract

Rare diseases (RD) pose a clinical, social, and ethical challenge: their low individual prevalence contrasts with a significant populatio level burden, prolonged diagnostic delays, a heavy family burden, and limited treatment options in most cases. The emergence of gene therapy and certain other advanced therapies, including those based on gene silencing or modification and other highly personalized (N-of-1) approaches—opens opportunities for patients with no alternatives, but increases clinical uncertainty and highlights gaps in our regulatory frameworks. Objective: To analyze, through a literature review and a normative bioethical analysis, the implications of biomedical innovation for therapeutic equity and human dignity in Mexico. Methodology: A documentary review of biomedical and bioethical literature, along with a comparative examination of international regulatory frameworks and applicable Mexican regulations. Results: Tensions are identified between beneficence and non-maleficence, autonomy and distributive justice, as well as specific gaps regarding exceptional access and the monitoring of highly personalized interventions. Conclusion: Mexico has relevant regulatory foundations, but requires explicit guidelines, streamlined procedures, and ethical safeguards that reduce inequality, prevent the provision of interventions without evidence, and promote responsible access to innovation.