Readers like you keep news free for everyone.

More than 5,000 readers have already pitched in to keep free access to The Journal.

For the price of one cup of coffee each week you can help keep paywalls away.

Support us today
Not now
Tuesday 5 December 2023 Dublin: 3°C

# Spinraza

All time
'I've can't lift my arms, brush my hair, or even go to the toilet alone': Children still waiting on Spinraza drug, Dáil told
The drug is used to treat SMA which can develop in babies and toddlers and can significantly reduce life expectancy.
Spinraza campaigners 'finally have hope' as HSE approves access to drug for rare disease
The drug has been approved for children with SMA Type I, II or III on “an exceptional and individualised basis”.
'We need to do right by families': Call for HSE to fund drug for rare disease after NHS approval
Spinal muscular atrophy weakens muscles and causes problems with movement, breathing and swallowing.
'We are failing these children spectacularly': Row over drug with 'exorbitant' cost rumbles on
Negotiations, and clashes, over the price of Spinraza are ongoing behind the scenes.
'Look in my child's eyes and tell him his life is not worth saving': Parents call for access to medication
About 26 children with SMA, a rare muscle-wasting condition, could benefit from Spinraza.
'A price has been put on the value of life': HSE will not reimburse drug for children with rare condition
The HSE said the current price of Spinraza – more than €20 million over five years – is not cost-effective.
'You wouldn't tell a child they couldn't have chemo, but our son is being denied the medication he needs'
Parents of children with a rare genetic condition are fighting for access to a potentially life-changing drug.
'If she gets another chest infection she probably won't make it, that’s the reality'
Nellie and Lara are the only two children in Ireland with Spinal Muscular Atrophy Type 1 – an extremely rare genetic condition.