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14-Month-Old In Race Against Time To Raise $2 Million For Life-Saving Treatment

by : Saman Javed on : 02 Apr 2021 16:42
14-Month-Old In Race Against Time To Raise $2 Million For Life-Saving TreatmentVerner Kjæsgaard/Charles Njuguna

Parents of a 14-month-old baby girl who has been diagnosed with a rare genetic disease are trying to raise $2 million for potentially life-saving treatment.

Ayah Lundt, who her parents describe as a ‘bubbly little princess’ has a rare genetic and degenerative disorder called spinal muscular atrophy (type 2).

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The disease affects the central nervous system, voluntary skeletal muscle movement and the peripheral nervous system, which connects the central nervous system to the rest of the body.

It currently affects one in 10,000 babies and has no cure.

Charles NjugunaCharles Njuguna

The adorable little girl initially developed as expected for a baby her age and hit all the usual milestones. She was diagnosed with SMA at ten months old after her parents noticed she was barely using her legs.

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The onset of SMA affects basic motor functions, such as walking, hand movement and feeding. Worryingly, as the condition progresses, patients lose their ability to swallow and breathe, causing death.

While there are treatments available to limit the progression of the disease, these must be taken up as soon as possible because the damage caused by the disease is irreversible.

Currently, Ayah is receiving a prescription drug, Spinraza, which is a spinal injection that is administered throughout a patient’s life.

Verner KjærsgaardVerner Kjærsgaard
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Her parents, from Denmark, are now in a race against time to raise enough money for her to receive a new, one-time gene therapy treatment called Zolgensma.

At $2.1 million a dose, it is currently the most expensive drug in the world. The treatment won’t cure the disease and can’t reverse any damage already done, but other children who have received the drug have been able to walk.

It is recommended that the earlier the patient receives the treatment, the better. Unfortunately, Zolgensma is only approved for children under two years old, which means Ayah’s parents have just 10 months to ensure they raise the funds.

Charles NjugunaCharles Njuguna
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‘Like any parent, we wish the best for our child, but even more so regarding her health. Besides Zolgensma only having to be administered once, it has proven to be the most effective in clinical testing,’ Ayah’s parents said.

They are now trying to raise enough funds for Ayah to travel to a Boston hospital and receive the treatment. Their goal covers the costs of the treatment itself, the consultation fee and travel expenses.

‘By giving Ayah this treatment, we hope to give her an almost normal childhood where she will be able to play with her big sister without any major obstacle,’ her parents said.

You can donate to Ayah’s treatment through GoFundMe here.

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Topics: Life, Boston, Denmark, gofundme, Now