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Matilda Donald’s ‘amazing’ transformation thanks to breakthrough drug

When Matilda Donald was 11-months-old, her parents were told she had a degenerative motor neuron condition and her life expectancy was slashed to just two years.The young girl was diagnosed with Spinal Muscular Atrophy (SMA), a rare inherited genetic disease that causes muscle weakness and wasting.Matilda was told she would never be able to walk…

When Matilda Donald was 11-months-old, her parents were told she had a degenerative motor neuron condition and her life expectancy was slashed to just two years.

The young girl was diagnosed with Spinal Muscular Atrophy (SMA), a rare inherited genetic disease that causes muscle weakness and wasting.

Matilda was told she would never be able to walk and her mother, Seona, described her diagnosis as “just horrid”.

But five days after being given the horrific news the toddler began receiving a breakthrough drug, Spinraza.

“And the treatment was just amazing,” Mrs Donald told reporters on Sunday. “It’s life-changing and in Matilda’s case, it was lifesaving.”

“Matilda had stopped rolling at around 10 months, but within two months of receiving treatment she was able to roll again and shortly after she could sing ‘Twinkle Twinkle Little Star’ with her hands well above her head.

“At the age of two-and-a-half, she took her first unsupported steps using parallel bars and in August last year she walked unsupported.”

And now, Matilda is an energetic four-and-a-half-year-old who is described endearingly by her mother as a “monster”.

Access to Spinraza will now be expanded through a subsidy from the Federal Government, meaning the treatment will be slashed from $367,000 a year to $6.60 per script with a concession card, Health Minister Greg Hunt said.

“The expanded listing of Spinraza will allow infants and children, diagnosed with SMA through a genetic test, to start treatment prior to the onset of symptoms,” he said.

“This means children and infants diagnosed with SMA will be able to commence treatment with this medicine from birth and up to three years earlier, which is so critical to address the impact of this debilitating condition.”

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