Why are biotech drugs relevant to orphan diseases?

Biotechnology provides powerful tools to develop diagnostics and treatments for orphan diseases. As most of the rare diseases are genetic, at least 80% of rare diseases have identified genetic origins. That is why medicines manufactured through biotechnology and gene technology can provide a solution to treat rare diseases. 

In gene therapy, genes are introduced to replace, repair or switch off defective ones. One example of a genetic disease is the cystic fibrosis which is already treated by gene technology.


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