NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, is joining the National Organization for Rare Disorders (NORD) and other organizations around the world today to observe World Rare Disease Day. In the U.S., the observance is led by NORD and focuses on the 25-30 million people living with a rare disease, many of whom have difficulty getting the right care because of a lack of education about these disorders.
This year also marks the 30 th anniversaries of NORD and the Orphan Drug Act. The legislation, which was driven by a determined group of individuals that also founded NORD, encourages the development and commercialization of treatments for rare diseases, or “orphan” drugs. The act was designed to provide financial incentives, including seven-year market exclusivity for an FDA-approved treatment and an accelerated review process for drug candidates that qualify. Shortly after the act was signed, NORD was officially formed and 30 years later, the organization is widely recognized as the leading advocate for awareness and recognition of the challenges faced by people living with rare diseases.
“Considering about one in every ten people in the U.S. is living with some form of rare disease and as many as 60 million worldwide, Rare Disease Day is an observance that truly transcends cultures and borders and is deserving of international attention,” said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. “We at NPS recognize NORD as a noble champion of patient advocacy in the rare disease community and applaud the organization for its 30 years of service.”
In the U.S., any disease affecting fewer than 200,000 Americans is considered rare. According to the National Institutes of Health (NIH), there are approximately 7,000 such diseases, yet less than 5 percent of these disorders have FDA-approved therapies.
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