Inherited metabolic disorders are a range of diseases caused by defective genes. Though rare, they can have devastating consequences for patients and their families. Research by the pharmaceutical industry has led to many specialised treatments. However, further improvements are still urgently needed.
Disease: Inherited Metabolic Disorders
Last update: May 2005
Intro
FAQ: What does the future hold?
Thanks to recent achievements in understanding the causes of metabolic disorders many new therapeutic approaches are now being investigated. These include the development of further enzyme and protein substitutes to enable the treatment of a wider range of these devastating diseases.
In the long term it is hoped to be able to isolate certain genes which have the potential to correct inherited disorders.
In the long term it is hoped to be able to isolate certain genes which have the potential to correct inherited disorders.