When you see Ayah’s pictures and videos on her Instagram, you will fall in love
with her sweet, lovely smile.
At first glance, you will not see a child fighting the rare genetic and degenerative neuromuscular disorder called SMA (Spinal Muscular Atrophy) type 2.
Ayah just turned one year old on the 15th of January 2021, and until she was nine months, she was developing like any other child of her age.
Shortly before her ten-month check-up, her parents noticed that, unlike her elder sister, Amira, Ayah struggled to do simple things she could do before.
Lifting a spoon to her mouth or standing, which would seem like a mundane task for a toddler, became increasingly difficult and frustrating for Ayah.
Concerned, her parents called their assigned home nurse.
A devastating diagnosis
After several medical examinations, Ayah was finally diagnosed with Spinal Muscular Atrophy type 2 (SMA-type 2).
SMA is a rare genetic disease that affects the central nervous system and voluntary skeletal muscle movement. It then progresses to affect the peripheral nervous system - the connection between the central nervous system and the rest of the body.
As a result, SMA affects essential motor functions such as walking, hand movements and feeding. After progression, the patient loses the ability to swallow, breathe and eventually die.
SMA affects one in 10,000 births and is a leading genetic cause of death among infants.
Currently, patients only receive treatment options to prevent the ravaging effects of SMA progression, as any damage caused by the disease is irreversible.
After her diagnosis last year November, Ayah has been put on Spinraza, a prescription drug that a patient gets throughout their life.
Spinraza strengthens the missing SMN2 gene (which is a backup of the SMN1 gene - the one that she is missing) to produce SMN protein, improving motor functions and movement.
Ayah’s family is grateful that she is undergoing treatment. In the last few months, Ayah has received four doses. All administered through the spine. The treatment is not only invasive but also a lifetime prescription.
There is hope in the world’s most expensive medicine
Through thorough research and consultation with experts, Ayah’s family is seeking a new and more effective but ultra-expensive drug called Zolgensma. The cost of the drug and treatment abroad comes to approximately 2.4 million USD (15 million Danish Kroner).
Zolgensma is a one-time-only gene therapy treatment for children aged less than two years. At 14 months old, Ayah’s window for treatment is slowly closing down.
The hefty price of Zolgensma is tagged on its ability to replace the missing SMN1 gene with a working copy that helps motor neuron cells work properly and survive.
Zolgensma is NOT a cure since it does not reverse the damage already caused by SMA before treatment.
Since Zolgensma is not yet approved for use in the Danish hospitals, the Danish healthcare system can only provide Spinraza.
SMA is a cruel disease. It makes you watch as your child’s muscular strength deteriorates, to the point that the child cannot breathe unassisted.
The clock is ticking, and Ayah’s parents cannot afford to wait until the Danish healthcare system finally approves treatment of SMA with Zolgensma in Denmark.
Therefore, it is up to them, through the support of the global and local community, to raise the money and seek treatment for their baby abroad. And which parent wouldn’t take this option if it means their child will have a shot at a normal, pain-free, and healthy life?
This fundraising campaign aims to raise the 2.4 million USD (15 million DKK) to cover the costs of Zolgensma, as well as travel, consultation and treatment fees in the United States of America.
By giving Ayah this treatment at an early age, her parents hope to provide her with an almost normal childhood. She will play with her big sister and live everyday life without any significant obstacle.
“We are in a race against time to achieve our goal since this treatment is currently only approved for children under two years old” emphasizes, Mary, Ayah’s mother.
The family invites people, organizations and interested parties to donate towards sending Ayah to the US and receive the treatment.
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