The families of children with spinal muscular atrophy (SMA) have led the fight to get access to Spinraza for all those who could benefit from it.
Muscular Dystrophy UK is calling for a temporary scheme for patients to access the treatment after NICE rejected Spinraza for funding on the NHS in initial guidance about the treatment.
Below we meet three families who are hoping to get access to Spinraza – and one family who have been able to get it on what a difference it makes.
“The one glimmer of hope we have is Spinraza”
Two-year-old Avery was diagnosed with SMA Type 2 in November and now requires 24/7 care.
He has a Wizzy Bug to support his mobility and a standing frame, and also uses a cough assist machine, a cough vest and suction machine.
Avery’s dad Alex says:
“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.
“The one glimmer of hope we have is Spinraza, and this news is such a blow to families like ours.
“It’s agonising to see your child slowly lose their mobility. But it doesn’t have to be this way. By approving Spinraza, NICE would throw families like ours the lifeline we so desperately need.”
“We would move heaven and Earth to secure a better future for Ayden”
Four-year-old Ayden was diagnosed just two weeks after his first birthday. His parents, Aliya and Khalil, first noticed something was wrong when Ayden was 11 months old.
Previously he had been able to stand whilst holding onto the coffee table, but suddenly he no longer could – his legs buckled beneath him, unable to support his weight.
Mum Aliya says:
“Spinraza may not be a cure, but it could buy many families more time to spend with their loved ones. We would move heaven and Earth to secure a better future for Ayden.
“Knowing there is a treatment out there that could make life easier for our little boy has given us renewed hope. NICE has the power to bring this important breakthrough treatment to the families who really need it. If NICE squanders this opportunity, families, like ours, will be made to pay the heaviest price.”
“It would give hope to families like ours”
In February 2017, aged one, Sophie was diagnosed with SMA Type 2.
Her parents, Raul and Tammy, noticed something was odd when she suddenly stopped jumping and refused to stand on her feet.
Mum Tammy says:
“When we heard that NICE was reviewing Spinraza, it lifted our spirits. But as more time passes without access to this treatment, the more Sophie loses the ability to do the simple things that other children take for granted.
“We know that Spinraza is not a cure, but it could give Sophie more independence, and she will have a better quality of life. If NICE approved this treatment, it would give hope to families like ours.”
“Getting access to Spinraza has been a lifeline”
Three-year-old William was diagnosed with SMA Type 1 as a baby. His parents were told that he was unlikely to live past his second birthday, but he has recently turned three. William – whose twin brother, Matthew, does not have SMA – has access to Spinraza through a temporary scheme organised by the drug manufacturer Biogen for those with SMA Type 1wor, and has done since August.
Since then, his head and hand movement has improved, as well as his breathing.
William’s mum Kerry says:
“We’ve had to fight hard, but getting access to Spinraza in August last year has been a lifeline. Although it is early days in William’s treatment, the improvements in his respiratory function have been significant. We’ve also seen increased movement in William’s hands, feet and head, all of which is going a long way to increasing William’s independence and communication”
“SMA is a cruel degenerative condition. We’ve seen the difference Spinraza has made to William in halting further deterioration and making huge improvements. For the first time we are hopeful for his future. We just hope NICE will make it available to all children who could benefit from it.”
Find out more about how you can help us fight for access for Spinraza.