rare diseases Latest News

First human trial for Tay-Sachs, Canavan and other rare enzyme deficiency diseases plans to begin in early 2013 thanks to NTSAD, the nation's oldest patient advocacy organization, which leads efforts to find a cure or treatment.

ACI introduces its Guide to Orphan Drugs and Rare Diseases: Maximizing Opportunities and Overcoming Stumbling Blocks in the Designation and Development Process.

A clinically challenging and under-studied subset of diseases affecting the lymphatic system and grouped under the disease spectrum lymphangiomatosis and Gorham’s disease is the focus of a special issue of Lymphatic Research and Biology.

Citing the existence of over 6,000 rare diseases Joff Masukawa, Senior Director, Government Relations and Public Policy, Shire details his role in orphan product development and explains the importance of early engagement.

Conference Brings Together Industry Leaders to Overcome Commercialization Challenges and Enhance Patient Access to Orphan Drugs

Campaign Brings Together Support from over 90 Corporations, Non-Profit Foundations, Research Institutions, Hospitals and Advocacy Groups; New Blue Jeans Denim Ribbon Unveiled To Promote Rare Disease Awareness

The Penn State Football Uplifting Athletes fifth annual Lift For Life® event was a tremendous success.