Approximately 80% of rare disorders are genetic, and most are caused by variants in specific genes. New therapies in development and under evaluation in clinical trials are increasingly being targeted to patients with specific gene variants. Eligibility for many clinical trials requires that patients know what gene variants they have. Patients and families are sometimes unaware that genetic testing can provide important information relevant to decisions about treatment options.
This video was made possible through an educational grant from Alltrna. NORD is solely responsible for the content and partnered with the National PKU Alliance for content development and with Osmosis from Elsevier for video production. Learn more about NORD at rarediseases.org
6 сен 2024
