Leeds North West MP Greg Mulholland has welcomed news that the Translarna drug has now been signed off by England's drugs approval body NICE.

Leeds North West MP Greg Mulholland has welcomed news that the Translarna drug has now been signed off by England's drugs approval body NICE.

[Pictured left: Greg Mulholland MP with Nic Bungay from Muscular Dystrophy UK, doing a petition drop at 10 Downing Street]

The first ever treatment to address the underlying causes of Duchenne muscular dystrophy has received a positive funding recommendation from the National Institute for Health and Care Excellence (NICE) and will be made available to eligible patients across the UK. The drug, Translarna, is proven to slow down the degenerative impact of this life limiting condition, keeping patients walking for longer and improving quality of life.

The news comes after the cross-party, multi-organisational #FundOurDrugsNOW campaign that Greg Mulholland has led over the last 18 months with charities, families and campaigners. The campaign has fought for treatment for three particular ultra-rare diseases to be made available through NHS England. The news around Translarna is the second major win for the campaign following approval for the Vimizim drug to treat Morquio syndrome.

Translarna will be subject to a Managed Access Agreement between NICE, NHS England and the manufacturer, PTC Therapeutics, making the drug available for an initial period of up to five years. This will allow for further gathering of evidence on the treatments' benefit.

Families and charities have campaigned long and hard for this treatment to be approved, lobbying the NHS, politicians and the Prime Minister David Cameron over the last year and a half. This announcement ends an agonising wait for a decision.

Duchenne muscular dystrophy (DMD) is a severe progressive genetic muscle wasting condition, affecting mainly boys and occurring in roughly 1 in 3500 male births. Around 2500 people live with DMD in the UK. Muscle deterioration is rapid, with children confined to a wheelchair by 12 years old. The heart and respiratory muscles are affected and even with expert medical intervention, people with DMD, on average, die in their late twenties. Translarna will be used to treat those 13% of patients whose DMD is caused by a nonsense mutation.

Commenting, Greg Mulholland said:

"This is fantastic news and something many families have long waited to hear. The #FundOurDrugsNOW campaign has worked hard to demand NHS England make available a treatment for Duchenne muscular dystrophy. Translarna is proven to be an effective treatment, and the longer those with the condition have to wait, the worse their condition gets. That they can now get their treatment is news they will welcome. I want to take this opportunity to pay tribute to the charities, families, fellow MPs and all other campaigners who have helped deliver this result. The #FundOurDrugsNOW campaign has now won treatment for two of the three ultra-rare diseases that we have focussed on- we intend to keep going until we have won treatment for all three."

Paul Lenihan MBE, CEO of Action Duchenne:

"We are so delighted this day has finally arrived. Action Duchenne have been working towards this moment for 15 years and have worked tirelessly with to get this particular treatment approved. This decision is a hugely encouraging sign that both NICE and NHS England have listened to the patient community, bringing hope to each and every parent and patient fighting this devastating disease. Whilst this drug will only benefit 13% of the population, we hope this decision will set a precedent for other therapies in the pipeline and ensure that more pharmaceutical companies and academics investigate this disease area. As a community we won't rest until there is a cure".