Batten’s disease is, mercifully, a rare occurrence in the UK.
Experts believe there are between 100 and 150 young people living with a diagnosis.
The symptoms are horrific and strike in childhood.
The first manifestation usually comes with visual impairment leading to blindness.
There are seizures, loss of speech and restricted mobility.
In some extreme cases the child will suffer hallucinations and memory loss.
We spoke to experts and parents about this horrible disease. The word they all used is devastating.
But finally there is some hope.
The disease remains incurable but there have been successful trials of the drug Brineura.
It stabilises children, improves quality of life and can add 20 years.
It is very expensive, costing £500,000 per child per year.
But it is a snip, an absolute bargain, when you are talking about the life of a child.
That must be balanced against the pressures on the NHS.
We are not advocating the withdrawal of this drug.
But why is it so expensive?
BioMarin, the US firm behind it, had revenue of more than a billion dollars in their last set of results.
They are the same company who denied access to a drug used to treat another rare disease PKU.
It seems this case is heading the same way.
We should be sending the strongest message possible across the Atlantic: Here we do not tolerate profiteering before patients. Neither should you.
