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.
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.
The drug is used to treat SMA which can develop in babies and toddlers and can significantly reduce life expectancy.
The drug has been approved for children with SMA Type I, II or III on “an exceptional and individualised basis”.
Spinal muscular atrophy weakens muscles and causes problems with movement, breathing and swallowing.
Negotiations, and clashes, over the price of Spinraza are ongoing behind the scenes.
About 26 children with SMA, a rare muscle-wasting condition, could benefit from Spinraza.
The HSE said the current price of Spinraza – more than €20 million over five years – is not cost-effective.
Parents of children with a rare genetic condition are fighting for access to a potentially life-changing drug.
Nellie and Lara are the only two children in Ireland with Spinal Muscular Atrophy Type 1 – an extremely rare genetic condition.