New medication available to NI sufferers of rare illnesses

Health Minister Michelle O’Neill revealed on Wednesday that what she called “innovative new drugs” would be made available for conditions including Atypical Haemolytic Uraemic Syndrome, mucopolysaccharidosis type IVa and Duchenne muscular dystrophy caused by a nonsense mutation.

The first is described by the NHS as causing severe inflammation of blood vessels, which can cause renal failure. Around 90 patients in England were being treated for the condition as of the end of last year.

The second is a skeletal condition which occurrs in one in every 200,000 to 300,000 people (according to the US National Library of Medicine).

The third is a type of muscle wasting disease, which the National Institute for Clinical Excellence says exists in six to nine newborn children in England each year.

The minister said: “I am delighted to announce the availability of these drugs to allow patients with very rare conditions to receive pioneering new treatments.

“Whilst there are only a handful of people affected by these conditions, the new drugs have the potential to make a real impact on their quality of life.”