New Muscular Dystrophy Drug Granted FDA Orphan Designation in US
Why are orphan drugs so important?
Simply put, because orphan drugs give a slither of hope to those who suffer from a rare disease. Currently, there are as many as 4,000 to 5,000 orphan diseases worldwide that do not have any kind of drug or treatment option. Every time a pharmaceutical company begins developing a specialized drug to treat one of these rare diseases such as OPMD, they are responding to a public health need. In other words, the companies are making a difference in the lives of those who need it.
