Baby Matilde, the infant who saw the nation raise €2 million to give her the chance of life-saving treatment is about to get it.
All the bureaucracy has been swept away to the extent that medicines authority INFARMED is actually authorising the ‘most expensive single-shot treatment in the world’ for another Portuguese baby at the same time.
As the country repeatedly heard when Matilde’s plight was first publicised, Zolgensma – produced by Novartis – is the only medication that offers what appears to be a complete cure for babies with Spinal Muscular Atrophy, type one.
It is a gene therapy, administered just once, that literally annihilates the root cause of the disorder, freeing infants from what would otherwise be a death sentence.
Last month Zolgensma was Matilde’s only hope, but apparently out of reach. Portugal had not authorised its use, and thus it was not paid for by the State.
Then came the incredible response from the country, raising over €2 million in a matter of weeks.
Now it is simply a question of waiting till Lisbon’s Hospital Santa Maria has Zolgensma on the premises and is ready to use it.
In the meantime, Matilde and the ‘mystery’ baby in line for this ground-breaking treatment both have to be shielded from germs so that they are healthy enough to receive it.
And the money raised, now that the State is said to be financing this new treatment? Matilde’s parents have said it will be going to “all the other Matilde’s” in Portugal who are suffering from this life-limiting disorder.