Fight to get free 'life-changing' drug almost over

Macaulay, 10, has the progressive muscle-wasting disease Duchenne Muscular Dystrophy (DMD)

A family say it is outrageous they have had to fight for access to a free drug that slows the progression of a muscle-wasting condition.

Givinostat was conditionally approved in December to help slow the progression of Duchenne Muscular Dystrophy (DMD), but was only available in Leicestershire and Swansea due to local NHS budget arrangements.

The Newcastle Hospitals Trust, which treats DMD patients across the North East and Cumbria, said it was "finalising arrangements" to make it available for those who meet strict eligibility requirements.

The family of 10-year-old Macaulay from Penrith, Cumbria, welcomed the news but have spent months of campaigning for access.

Usually affecting boys in early childhood, DMD is one of the most common and severe forms of muscular dystrophy, which gradually causes the muscles to weaken, leading to an increasing level of disability.

The drug was initially offered for free under an early access programme, while it awaited full NHS approval.

However, health bosses had to fund the staffing for treating patients, blood tests and collecting data.

Macaulay's mum Rebecca has been campaigning, getting political support and writing to Newcastle Hospitals Trust to get access in the region.

Macaulay's parents Ben and Rebecca have campaigned for access to Givinostat for months

Rebecca said the drug would give her son a "longer, more independent life".

Macaulay, who loves sports, said he has had to cut back as the genetic disease progresses.

"I don't think I can run as fast as I used to because I'm on steroids," he said.

His dad Ben said the continued wait was frustrating.

"It's confidence, it's the ability to keep up with his peers for as long as possible, the ability to walk for him is really important," he said.

In a statement, the trust said: "We are pleased to confirm that we will be able to make Givinostat available through an early access programme to eligible patients in our clinics very soon."

Its clinical team would discuss the drug with patients who are potentially eligible at their next review appointments, the trust added.

However, Rebecca criticised the lack of timeline commitment, adding: "Yes, 'It will come soon', but what does soon mean?"

Co-founder of charity Duchenne UK, Emily Reuben, said it was "time critical" as the drug was "only available for boys who can walk".

"Some boys who were eligible for this drug six months ago are no longer eligible, because they've stopped walking," she said.