Posted by Michael Wonder on 09 May 2018
Rare diseases: addressing the challenges in diagnosis, drug approval, and patient access
9 May 2018 - Rare diseases represent a wide range of disorders and constellations of clinical signs and symptoms.
Many rare diseases cause chronic or progressive physical deterioration, disability, or premature death and start in childhood, creating a huge burden on parents and caregivers. Most rare diseases are thought to be genetic and there may be as many as 7000 rare diseases.
Although there is no universally accepted terminology or definition as to what constitutes a rare disease, it is typically characterised by its low frequency of occurrence.
