Six children, including a 6-year-old Indian-origin boy, have been denied life-saving treatment from a rare disease due to the high costs by UK's state-funded health service. Kirath Mann, 6, suffers from Duchenne Muscular Dystrophy (DMD), which affects one in 3,500 boys in Britain and leaves several of them in wheelchair before their 10th birthday.
Kirath’s family passionately campaigned with other families and end delays to the treatment called ‘Translarna’ but they reportedly failed and naturally felt devastated. "We are devastated at having to face yet more disappointment after another hurdle has been put in our way. All these organisations and National Health Service (NHS) bodies that have been involved in the decision do not seem to be appreciative that this is a rare disease and very progressive one as well", said Kirath's mother Jaspal.
The children with the condition including Kirath even wrote ‘personal’ letters to the British PM David Cameron in a bid to get the drug that could save their lives. "Hello Prime Minister, my name is Kirath. I am six years old. My legs are poorly. Please help me get the medicine I need," read the letter in his childish scrawl.
The drug, also known as ‘Ataluren’ is the first-ever to tackle the causes of DMD and was approved in Europe in August 2014, is available in France, Spain, Germany, Italy and Denmark, but not in the UK. The National Institute for Health and Care Excellence (NICE) is reportedly deciding on commissioning of the new drug and decided on requiring more information on the benefits of the drug, which can cost 4 million pounds per patient per year.