By 2019-06-28 InPortugal
 

Battling baby Matilde “in race against time”

SEE UPDATES BELOW: The €1.9 million has been raised ‘and some’:

Huge wave of solidarity raises €2 million euros for sick baby’s miracle treatment

A heartwarming wave of solidarity has swept the country raising two million euros for the ‘one-shot’ treatment of the ‘most expensive medication in the world’ that may bring baby Matilde Sande the chance of a normal life.

The campaign mounted by the parents of the two month old reached exceeded its objective by over 100,000 euros on Tuesday, as Matilde herself succeeded in battling a respiratory infection and was on track for leaving intensive care.

What happens next is what everyone is waiting to find out.

Reports explain, Matilde is possibly not well enough to make it to America, where Zolgensma, the ‘miracle drug’ that has saved 19 babies like her, is available to those who can pay for it.

AveXis, – a subsidiary of Novartis –that produces Zolgensma, has been in touch with Matilde’s parents and the Portuguese Neuromuscular Association.

What is hoped is that an ‘exceptional case’ may be made by the European Medicines Agency so that Matilde can receive Zolgensma in Portugal.

This story has shone a much-needed spotlight on SMA (spinal muscular atrophy) and the youngsters that suffer with it.

Joaquim Brites of the Portuguese Neuromuscular Association has explained that there are nine other ‘Matilde’s’ in Portugal – all of them living in the shadow of a ‘death sentence’.

The only drug authorised in Portugal (and the rest of Europe) is Spinraza – administered through ‘various injections’ and acting on the defective gene that causes SMA, staving off its effects.

Zolgensma’s advantage is that it is described as ‘deliverying a healthy copy of the gene that has been damaged’ by the disease, allowing patients the chance of a completely normal life.

By coincidence perhaps in the wider sphere Spinraza’s producers Biogen are in a major competitions war with Novartis and also with Roche.

Earlier story…

Baby Matilde – the little girl who needs one shot of the most expensive medication in the world (click here) – has had to be readmitted to hospital with breathing difficulties.

Her parents, a young couple from Oeiras with two other chilren, have admitted they are in a ‘race against time’.

Their fund-raising campaign has amassed just under €400,000 – but that leaves €1.5 million to go (see update below).

As TV personality Manuel Luís Goucha said earlier this week, if all of us gave just €1 the family could be on their way to America for the life-saving injection tomorrow.

Meantime, the baby is undergoing the “available treatment in Portugal”, which can only guarantee to prolong her life by two or three years.

Matilde’s condition, Spinal Muscular Atrophy type 1 – described as the worst form of the disease – is one shared by just over 100 people in Portugal, 80 of them children.

A one-shot dose of Zolgensma is her only hope. Said mother Carla this week “results have been fantastic in the babies that have taken it. They are all healthy and don’t depend on machines”.

But today, Matilde is on a machine, receiving assisted ventilation and being treated with antibiotics for a blocked lung. Her parents are just hoping for a miracle.

Anyone who would like to contribute to the campaign to help save Matilde can do so by making a donation to the account of Matilde Sande on PT50 0035 0685 00008068 130 56.

For full details, see the “Matilde, uma bebé especial” Facebook page.

UPDATE MONDAY JULY 1:

The campaign has raised over 1.2 million euros! Matilde’s parents have written that they are getting “closer and closer” to the end goal, and hope that their little daughter can be given a running chance at life.

Meantime, reports in the wider press have suggested Zolgensma -‘the most expensive treatment in the world’- could become available in Portugal by the end of this year.

For a full update, see our paper edition out on Thursday.

natasha.donn@algarveresident.com


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