| Popis: |
Approximately 25 million Americans and many more throughout the world suffer from more than 6000 rare diseases. Some of these diseases are wellrecognized, such as Lou Gehrig’s disease or cystic fibrosis, but little is known about many others, such as Adams-Oliver syndrome or Norrie disease. Almost all of them are serious, life-threatening, or fatal diseases. Despite the urgent need for safe and effective treatments, the small patient populations of these rare diseases often do not present sufficiently viable markets for drug sponsors to recover the high costs of therapeutic research and development, much less to expect profit. After investing resources to develop the drugs, the sponsors may not be protected from competitors since the potential uses of many drugs in rare diseases—often discovered during the course of study for other diseases and generally already in the public domain—may not be patentable. Thus, even after being discovered, many of these drugs are not commercially pursued or “adopted.” Hence, they come to be known as “orphan” drugs. |
