Rare Disease Day 2013: Rare Disorders Without Borders

February 28, 2013 | by

Today is the sixth annual Rare Disease Day, an international advocacy day held on the last day of February — a rare day for rare people. On this day, millions of patients and their families from more than 60 countries and regions around the world will share their story to promote awareness of the challenges of living with a rare disease and bring widespread recognition of rare diseases as a global health challenge. This year’s slogan is “Rare Disorders without Borders”, which emphasizes the need for international cooperation.

Rare diseases without borders

Trade Group Study: Hundreds of Rare Disease Drugs in Development

February 21, 2013 | by

According to a new report released last month by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA), the biopharmaceutical pipeline is innovative and robust, with a high percentage of potential first-in-class medicines (meaning a new treatment where nothing currently exists) targeting diseases with limited treatment options. In addition to identifying medicines in development for conditions and diseases such as septic shock, ovarian cancer, sickle cell disease, and Lou Gehrig’s disease (amyotrophic lateral sclerosis), which haven’t had any new product approvals in the last ten years, the report offers positive news for the rare disease community: one third of the products currently in clinical development have a rare disease designation by the U.S. Food and Drug Administration (FDA).

PhRMA

NIH Common Fund Announces New Programs

July 2, 2012 | by

New programs exploring novel approaches to cell-to-cell communication and understanding undiagnosed diseases, which represent challenges or scientific opportunities for a wide array of health research, are the latest priorities for the National Institutes of Health Common Fund. The funding was announced today by NIH Director Francis S. Collins, M.D., Ph.D., after he received broad community input and recommendations from institute and center directors at NIH. The Common Fund targets strategic investments that have the potential for rapid and significant impact. The programs are scheduled to begin during fiscal year 2013.

NIH Common Fund