Research in rare diseases has contributed substantially toward the current understanding in
the pathophysiology of the common diseases. However, medical needs of patients with rare …

Finding ways to bring new therapies for rare diseases to patients in a timely manner,
effectively and affordably is an important public health challenge. The key concern for …

Purpose To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers
and discuss possible reasons for the small number of products licensed. Methods …

We are pleased to announce the launch of Orphanet Journal of Rare Diseases, an open
access, peer-reviewed journal that will encompass all aspects of rare diseases and orphan …

Increasing the use of orphan drugs in clinical practice Page 1 Orphan drugs enable people
suffering from rare diseases to access well-tol- erated, effective drugs. This article reviews …

Orphanet is a European initiative that aims to improve the management and treatment of
rare diseases. It comprises a database dedicated to information on rare diseases and …

Orphan drugs (ODs) are products developed for the diagnosis and/or treatment of rare
diseases and conditions. Patients with this group of disorders have historically been denied …

Despite the existence of numerous rare neurological diseases, no studies have been
conducted on orphan drugs for neurological diseases available on the Japanese …