Babies with a rare genetic condition will needlessly die unless a drug branded a miracle is urgently approved on the NHS, charities have warned.
Spinraza is proven to reverse spinal muscular atrophy, which kills most sufferers battling the most severe form before they reach their second birthday.
Health officials have been locked in negotiations for over a year in trying to get the drug - estimated to cost £450,000 per patient per year - made available on the NHS.
Now, Muscular Dystrophy UK and Spinal Muscular Atrophy UK are demanding urgent action to break the deadlock and allow doctors to dish out Spinraza.
Rachel Jamieson’s four-year-old daughter, Matilda, has SMA Type 3. This form of the condition does not affect life expectancy, but leaves children less able to stand and walk over time
Catherine Woodhead, chief executive of Muscular Dystrophy UK, said: 'The continued delays are unacceptable.
'The stark reality is some children will die without access to Spinraza.' The drug is made by Biogen, a Massachusetts-based pharmaceutical firm.
Ms Woodhead said no more time can be wasted, adding: 'This is devastating for families, who know there is a treatment that could help but is agonisingly out of reach.
'In the 16 months it has taken for this drug (also known as nusinersen) to be assessed, lives may needlessly have been cut short.'
Spinraza is proven to reverse spinal muscular atrophy, which kills most sufferers battling the most severe form before they turn two
She added that, in the meantime, children and adult sufferers may also have lost the ability to walk, breathe and swallow because of there being no approved drug.
Ms Woodhead added: 'The Government must recognise the system is broken and urgently act to overhaul the appraisal process for treatments for rare diseases.
'But right now, NICE, Biogen and NHS England must end families' heartbreak by showing flexibility on cost and coming to an agreement urgently.'
Spinal muscular atrophy is a genetic condition which causes nerves and muscles to deteriorate. It affects around 1,300 Britons.
The worst form of the muscle wasting condition – type 1 – affects one in 10,000 children and kills them all before their second birthday.
Spinraza works by fixing the faulty gene, causing the body to read DNA differently and produce more of the proteins needed to build nerve cells.
As a result, muscles grow stronger and nerves work better, meaning the condition doesn't always progress.
SMA symptoms include floppy or weak arms and legs, difficulty sitting up, crawling or walking, swallowing problems and breathing difficulties.
A mother has today said it is 'unthinkable' that patients with spinal muscular atrophy are still unable to access Spinraza.
Dr Sheonad MacFarlane's daughter, Eilidh, 10, has SMA type 2. She is able to get the drug because it is approved for use in Scotland.
However, Dr MacFarlane, from Glasgow, said her heart goes out to families in England, Wales and Northern Ireland who can't the drug.
She said: 'I just hope NICE can reach a decision soon.
'Having access to Spinraza means so much to us and to other families; we are overjoyed that Eilidh now has access to this treatment.
'We still don’t know what the future holds, but we, at last, have hope. As a parent, it’s heart-breaking to see your child gradually lose their physical abilities.
'Nothing can prepare you for the devastating emotional impact this has on you, particularly when there is a treatment out there.'
Dr Sheonad MacFarlane's daughter, Eilidh, 10, has SMA type 2. She is able to get the drug because it is
