Meeting the needs of patients with ultrarare diseases.

Abstract excerpt

Patients with ultrarare diseases present unique challenges to the health care systems of developed economies that demand novel approaches, beginning with achieving a diagnosis and concluding with long-term treatment. The challenges derive from numbers. On the one hand, the rarity of the disease phenotypes means that the vast majority of ultrarare patients are never diagnosed, and for the fortunate few who are...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Delivery of Health Care
• Share to Genome, Human
• Share to Phenotype