Rare Diseases & Orphan Drugs

Biomarkers for Rare Disease Diagnosis and Treatment

Biomarkers are crucial in the diagnosis and treatment of rare diseases, providing objective measures to assess disease progression and response to treatment. They can also be used to identify patients who may benefit from specific treatments, and to predict treatment outcomes. In rare diseases, where symptoms and progression can vary widely, biomarkers can aid in the accurate diagnosis and monitoring of the disease. Additionally, biomarkers can help in the development of new treatments by providing information on disease mechanisms and potential targets for intervention. As such, biomarkers play a critical role in the advancement of rare disease research and the development of effective treatments for patients.