How Do Molecular Mechanisms Inform Pediatric Therapeutics?
Targeted therapies based on molecular mechanisms are revolutionizing pediatric care. For example, enzyme replacement therapy is used to treat lysosomal storage disorders like Gaucher disease. In oncology, targeted inhibitors such as those against the BCR-ABL fusion protein in pediatric leukemia have significantly improved outcomes. Personalized medicine, which tailors treatment based on an individual's molecular profile, is becoming increasingly feasible and effective.
