Rare diseases, also known as orphan diseases, are those which affect a minority of the population. Seven thousand rare diseases have been identified to date and affect 3.5 million individuals in UK, and 300 million globally.
Almost half of the known rare diseases are enzyme disorders – the human body either producing a deficient version of a given enzyme or failing to produce it all.
As many of these diseases have only been identified in the last few decades and have relatively small patient populations, it is perhaps not surprising that the pharmaceutical industry has yet to produce effective treatments for the majority of them.
Protein Technologies Ltd. is a world leader in the area of drug development for rare or neglected diseases which it addresses by means of drug repositioning or enzyme replacement therapy.
Currently the company is working in collaboration with a number of clients to develop effective therapies for certain rare diseases.