Little Lara and Nellie have a very rare neuromuscular condition.
SMA stands for Spinal Muscular Atrophy and is genetically inherited. It affects the ability to walk, arm, hand, head and neck movement as well as swallowing. Children diagnosed with this terrible condition typically have a life expectancy of two years.
But a drug was developed that has been successful in treating it.
Nusinersen is the only treatment available for SMA type 1 – and it was the only hope for these two little girls.
Their families have been campaigning over the past few weeks to get the drug approved here in Ireland. Time was against them as their daughters were at risk of rapid deterioration.
Happily, we can bring you the news that both Lara and Nellie have been given access to this ‘wonder’ drug and will start treatment in Temple Street Children’s Hospital within the next few days.
(Here’s Nellie playing with Olaf from Frozen on the CureSMA Facebook Page)
Nellie’s dad, Stephen says they will now be able to focus on getting Nellie off her breathing machine and is hopeful she will be able to get her swallow back which will make a huge difference to her little life.
Both families say they are overwhelmed with the decision. They are hugely grateful for all the support they received. They believe it is because of people power and the willingness of others to drive their campaign forward that they were so successful.
We wish Lara and Nellie every success with their treatment.
