The mum of two boys who both suffer from the same deadly disease has spoken of her heartbreak and relief after one son was approved for a £1.8 million wonder drug on the NHS, and the other was denied it.
Two-year-old Rocky Smith was born with severe spinal muscular atrophy (SMA) before brother Bentley, seven-months-old, was also diagnosed with the same condition in a cruel twist of fate.
The rare and often fatal genetic disease occurs once in every 10,000 births and causes paralysis, muscle weakness and progressive loss of movement.
However, because the drug hasn't been trialed on children aged two and above, whilst Bentley will receive the drug Rocky will not.
Mum Justine Smith, 26, has now described both her joy and heartache after seven-month-old Bentley was given the go ahead to receive a potentially life-saving treatment.
But Rocky could face an entirely different future to his sibling as he does not qualify for the same treatment - because he is too old.
The pioneering gene therapy was recently made available after NHS England announced it had struck a deal in March with manufacturer Novartis Gene Therapies.
On July 8 Bentley became one of the first people in Britain to have a dose of Zolgensma - which costs a staggering £1.79 million a dose.
The treatment - believed to be the world's most expensive drug - is given as a single intravenous infusion and contains a replica of the missing gene SMN1.
Justine said Bentley is already showing signs of improvement since being treated as Bristol Children's Hospital, one of only four treatment centres nationwide.
But she also spoke of her devastation at not being able to give Rocky the same chance of hope.
Babies born with severe type 1 SMA - the most common form of the condition - have a life expectancy of just a few years.
Until two years ago there were no treatment options for children with the condition, which is the leading genetic cause of death for youngsters.
Justine, from Upton-upon-Severn, Worcs., said: "It has been really hard. The condition is rare anyway but for both of them to have it just doesn't really happen.
"You are not meant to have another baby with SMA but somehow I managed it. It is such a slim chance, and they have the most severe type too.
"I'm part of a Facebook group for parents with kids with SMA and there are only about 30 in the UK with it.
"Since Bentley had the drug, we have noticed a few improvements in him already.
"He has a lot more head control, more movement in his arms and he is kicking his legs. It has made quite a bit of difference already.
"He has to be constantly monitored for four months and have regular blood tests because it can have bad side effects like kidney damage.
"At the moment his bloods are abnormal and his platelets are low which is causing him to come out in tiny bruises on his arms.
"The doctors said it is what they would expect if it was working. It is absolutely amazing really.
"I think it is one of the most expensive drugs in the world, we are incredibly lucky to have it.
"There are kids in America who have had it and they are now 15 and 16 when they would have been lucky to reach two.
"I would love my other one to have it, but it has not been trialled on over-two-year-olds, only on kids 12 months on under.
"It was hard to take when the doctors said he couldn't have it. It was gutting. So it's bittersweet emotions really.
"It has been heartbreaking but we're grateful for the chance for Bentley to have it.
"I know they are looking at doing trials on over-two-year-old's but if I gave it to Rocky and it didn't work he would deteriorate.
"The thing is with Rocky is that he is so weak and has been in and out of intensive care for three weeks with common cold and flu.
"He had to have chest physio and got influenza and ended up in intensive care at Birmingham's Heartlands Hospital.
"His hip on the left side was dislocated. If the common cold can make him like that then the treatment might make him even weaker. It just wouldn't be worth the risk."
Justine found out Rocky had SMA when he was rushed into Worcestershire Royal Hospital at three months old in September 2019.
She added: "They thought he had bronchitis and they treated him with antibiotics because didn't know he had SMA.
"His lung collapsed and he was in intensive care for three months.
"I was talking to other parents of children with SMA - Rocky is one of the worst, he is in and out of hospital all the time."
A genetic test at Royal Stoke University Hospital revealed Rocky had the rare condition while Bentley was later diagnosed while still in the womb.
Justine added: "Because of Rocky when I was pregnant with Bentley they tested him and found he had it too.
"The chances are just so slim. My eldest, my daughter doesn't have anything at all."
One of their boys' five consultants told her Zolgensma had become available on the NHS when she was eight months pregnant with Bentley.
She added: "I told them straight away I wanted him to have it.
"The drug was approved when he was born but he had to wait until July until the treatment centres were ready.
"I think it should be available worldwide and I would 100 per cent recommend it to other parents of children with SMA."
Neither Justine or husband Rocky, 34, who also have eight-year-old daughter Lexie, work because their boys require around the clock care.
The family have tried to raise funds to take Rocky Jnr to the US for gene therapy which would've cost $2.5 million but he was deemed too ill to fly.
Because Rocky Jnr falls outside the National Institute for Health and Care Excellence's (NICE) recommendation criteria for Zolgensma, he has been receiving with alternative Spinraza, which he is said to be responding well to.
Justine is supporting a campaign for screenings for SMA during pregnancy as, if treatment is delivered at birth, children have the best chance of long and healthy lives.
The petition can be found here.
