Gaucher disease is one of the more common of the lysosomal disorders; it quite commonly affects adults as well as children.

The adult form usually starts in childhood and causes enlargement of the liver and spleen, painful conditions in the bone, anaemia, and a tendency to bleed.

In very young children it can also affect the brain, starting off with a simple squint, but sometimes progressing to a horrible form of epilepsy in adolescence or later childhood.

Nobody knows why this occurs, but gene therapy seems to offer some prospect for improvement, as does enzyme therapy delivered to the brain.

Donations to a research initiative looking into the causation of the brain condition associated with Gaucher disease are invited and would be well used.

Gaucher disease is a less neglected area, and although some companies working in this very rare (ultra-orphan) field of medicine have shown interest in it, commitment from the public by a generous donation would indicate to them the need to move fast and step up their efforts with scientific doctors and researchers to develop corrective therapies.

Thank you sincerely for considering making a donation to this research; the human and scientific need is immense, but just now significant breaks are on the horizon.