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Hope for patients with rare diseases as new drugs to be made available on the NHS

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By Victoria O'Hara

Published 21/07/2016

Drugs for people with a range of rare diseases are to be prescribed by the health service, it was announced yesterday
Drugs for people with a range of rare diseases are to be prescribed by the health service, it was announced yesterday
Christine Collins, NIRDP chair
Minister Michelle O’Neill

Drugs for people with a range of rare diseases are to be prescribed by the health service, it was announced yesterday.

The move by Health Minister Michelle O'Neill was heralded as "wonderful news" and a development that would see people prescribed previously unavailable medication.

The drugs, evaluated under the Highly Specialised Technology Programme of the National Institute for Health and Care Excellence (NICE), will be given to patients with conditions including Duchenne muscular dystrophy and Morquio syndrome.

It is estimated that around 106,000 people in Northern Ireland live with rare diseases.

Some 10 boys are believed to be affected by Duchenne muscular dystrophy, a genetic disease that causes weakness and loss of muscle function.

Patients with the condition lack dystrophin, a protein found in muscles that helps to protect them from injury.

Among the drugs to be made available on the NHS is Translarna -a new treatment specifically for people with Duchenne muscular dystrophy.

"Whilst there are only a handful of people who are affected by these conditions, the new drugs have the potential to make a real impact on their quality of life," the Sinn Fein Health Minister said.

The patients who benefit the most from Translarna are those aged five years and over, and who are still able to walk and do not rely on a wheelchair.

The minister said that while the new medications were highly expensive, that did not mean that patients in Northern Ireland should be barred from accessing medical treatment that could dramatically improve their quality of life.

She added that it was important that the health service worked efficiently to improve people's lives.

"That means difficult conversations and decisions ahead," the minister explained. "It also means supporting patients to make the right choices in looking after their own health and wellbeing.

"It means that clinicians and other professionals must be certain that a drug, treatment or a service is the best and most effective option for their patient or client on the basis of all the information and evidence available to them."

Christine Collins, chair of the Northern Ireland Rare Disease Partnership (NIRDP), praised the development.

"This is a major step forward," she said. "NIRDP's vision is that in Northern Ireland no one is disadvantaged because of the rarity of their condition.

"We look forward to working with the department to make sure that all the systems are in place so that people in Northern Ireland can benefit fully and benefit quickly. This is about transforming lives by ensuring equality of access."

Dr Fiona Stewart, a consultant in genetic medicine, added: "It is wonderful news that patients with Morquio disease in Northern Ireland will now have the same access to treatment as patients in other parts of the UK.

"This means they are being treated in a fair manner and is great news for all affected."

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