A COUPLE’S hopes that a new treatment could help their five-year-old child to walk have been dashed by the decision by regulators not to approve the drug for NHS use.

Dominic and Tammy Wright, from Darwen, believed Nusinersen could radically improve their son Leyton’s muscle-wasting condition.

He suffers from spinal muscular atrophy which has left him unable to crawl, let alone walk, and confines him to a special wheelchair when he goes out.

They had hoped that dramatic results from the use of the drug, also know as Spinraza, in the United States and Europe would see it prescribed on the NHS.

Earlier this week the National Institute for Health and Care Excellence (NICE) ruled that it was too expensive and its long-term benefits were too uncertain to recommend its approval.

Each dose costs £75,000 and four are required in the first few months of treatment with regular follow-up doses every four months.

Mr Wright said: “This is the first-ever treatment to have any effect on Leyton’s condition.

“There are children with the most serious variant of the disease, Type One, that since being on Spinraza have taken their first steps.

“Leyton has the less serious Type Two and we believe this drug could help him to walk in future. He cannot even crawl at the moment.

“Our hopes were raised when we heard about the drug but NICE’s decision has dashed them.

"It was a real kick in the teeth.”

The couple are to write to Darwen’s MP Jake Berry and attend a demonstration outside NICE’s Manchester offices next month.

Mr Berry said: “This drug could be life-changing for Leyton but it seems NICE have concerns about its long-term effectiveness.

“I shall work work with the Wright family and NICE to see if there is a way forward to address these concerns.”

Meindert Boysen, NICE’s technology director, said: “The committee accepted SMA is a rare and very serious condition that can have a severe impact and that there is an unmet need for effective treatments.

"The very high cost of Nusinersen meant we could not recommend it as a cost-effective use of NHS resources.

“Nusinersen is a promising treatment that has been shown to improve a range of outcomes important to patients.

"We are actively engaging with Biogen to discuss how they might address the uncertainties identified while demonstrating the potential for Nusinersen to be considered cost-effective.”