Genetic Treatment Approaches in Rare Pediatric Diseases

Gene and cell therapies have been developed and approved for a growing number of pediatric diseases, with ongoing research for additional treatments. Each therapy is tailored to the specific disease and targets a specific genetic alteration or cell population. The development of new therapies is a complex and regulated process, with strict oversight at regional and European levels. Research begins in the laboratory and progresses through clinical trials to ensure safety and efficacy. Scientists are exploring novel therapies using human cells and tissues to model pediatric diseases. Clinical trials are conducted to evaluate the effectiveness of these therapies, and researchers are also investigating repurposing approved treatments for other diseases or aspects of the same disease.