A decision by UK health officials to reject a drug that treats a rare genetic disorder which can mean affected children do not live to their second birthday has been condemned by a charity.
In new draft guidance, the National Institute for Health and Care Excellence (NICE) has not recommended the drug Spinraza as a treatment for sufferers of spinal muscular atrophy (SMA) for widespread NHS use.
Muscular Dystrophy UK says the decision will cause heartbreak for families.
It says without access to the treatment children with SMA - which affects up to 2,500 people across the UK - will be unable to crawl, walk and swallow.
NICE says it welcomes further discussions about access to the drug, also known as nusinersen, manufactured by Biogen, saying while it showed substantial benefits, uncertainties about its long-term benefits remain.
Cost was also listed as an issue.
The condition affects the nerves in the spinal cord which control movement. This causes muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.
Spinraza is the first treatment to address the cause of motor neurone degeneration in SMA - it is injected directly into the spine and is a lifelong treatment.
Muscular Dystrophy UK, along with SMA Support UK and the SMA Trust, called for a temporary scheme so patients can access the drug while evidence is collected on its long-term benefit.
"Spinal muscular atrophy can be devastating and today's news will be heartbreaking for the families of those living with the condition," Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said.
"Once again we are seeing families suffer due to the appraisal process being too limited to assess costly but life-changing rare disease drugs.
"The one glimmer of hope is for a temporary scheme that ensures access."
Avery Ball, two, from Braunston, Northamptonshire, was diagnosed when he was 18 months old.
His father Alex said: "SMA is relentlessly cruel, and it turns your life upside down. To hear that your child will never be able to walk, or will struggle to sit independently, is heartbreaking. All your hopes, dreams and plans for the future are completely thrown off course.
"This news is such a blow to families like ours."
Australian Associated Press