There have been several successful examples of therapeutic target identification in pediatric diseases:
Acute Lymphoblastic Leukemia (ALL): Targeting the Philadelphia chromosome with tyrosine kinase inhibitors has significantly improved outcomes in pediatric ALL. Cystic Fibrosis: Modulators targeting the CFTR gene have transformed the treatment landscape for cystic fibrosis, particularly in children with specific mutations. Duchenne Muscular Dystrophy (DMD): Gene therapies targeting the dystrophin gene are showing promise in early trials.
