A family from Colchester is considering moving to France to ensure their two young sons who suffer from muscular dystrophy can obtain the drug they need to combat the disease.
Rob and Clare Baker said they would relocate if the drug Translarna, needed by their sons William, six, and Isaac, three, is not made available on the NHS.
Both suffer from Duchenne muscular dystrophy (DMD), which usually confines sufferers – most commonly boys – to a wheelchair by the age of 11 and leads to early death. Translarna is the first treatment to protect boys from the most serious effects of the disease.
The drug was granted conditional approval by the European commission in August 2014 and and is available in several countries including France, Italy and Spain.
Nice, the body that decides what drugs are available on the NHS, is yet to make a final decision about Translarna, which can cost as much as £200,000 per patient a year. A spokesperson for Nice said it was requesting more information from the US-based manufacturer PTC Therapeutics about the clinical benefit of the drug and its cost-effectiveness. A final decision is not expected until early next year.
Rob Baker said William was still able to walk but his mobility was deteriorating daily. “We are not sure how quick the onset will be. If he stops walking, Translarna will no longer be available to him,” he told Mail Online. “Isaac would still be able to get the drug at the same time as William because children with a sibling requiring the treatment are given access to it earlier.”
If Nice does not approve Translarna, he said he was prepared to move his family to France and commute to his job as a tax adviser in London, or spend weeknights away from his family. “Everything is on hold until we know. It’s going to be a sad and anxious Christmas,” he said.
The Baker family has been working with Muscular Dystrophy UK to lobby Nice, NHS England, ministers and MPs to win approval for the drug.
Jaspal Mann, whose son Kirath, six, also suffers from the condition, said earlier this year: “Translarna makes us feel that there may be some light at the end of the tunnel. It would buy precious time for Kirath just to be himself.”
