A COMPREHENSIVE REVIEW OF TREATMENT APPROACHES FOR CONGENITAL AND HEREDITARY ANOMALIES

Abstract

Congenital and hereditary anomalies (CHA) are functional/structural deformities that may be present at birth or develop later in life, potentially leading to significant health problems globally. Prevalence is high in low- and middle-income countries and causes a large number of mortalities annually, necessitating treatment interventions for effective management. The objectives of this study were to highlight various treatment methods for CHA and to evaluate the effectiveness of advanced medicinal therapies, such as stem cell therapy, nanomedicine, and genome editing, in controlling associated diseases. Diagnosis of CHA has been significantly improved through initiatives like newborn screening programs, which enable the early detection of abnormalities in affected infants. The treatment of CHA varies depending on the type of disorder and the specific needs of the affected individuals. For structural disorders, surgery is often recommended. Additionally, CHA can be addressed through stem cell therapies, medications, gene therapies, neurochips, and CRISPR/Cas9 gene editing. Medical interventions for CHA differ greatly based on the type and severity of the disorder. For example, steroids are commonly prescribed for muscular dystrophy, while physical therapy is beneficial for maintaining muscle strength and reducing weakness. Speech therapy plays a crucial role in developing communication skills in children. Hematopoietic stem cell transplantation (HSCT) is often recommended for various blood cancers and diseases. Furthermore, advancements in technologies such as CRISPR-Cas9 and improvements in prenatal and neonatal care have significantly enhanced  survival rates among children with CHA. Despite advancements in treatments, further research is still needed to overcome certain difficulties and treatments.