Those who are diagnosed with type 2 of SMA live between eight-to-10 years whereas ones with type 1 of SMA, its rarest form, do not live until the age of three.
Qatar Charity stepped in to gather donations for Nader, a three-year old Iraqi child with Spinal muscular atrophy (SMA), in hopes of securing $2.1 million treatment for his disease.
The Qatari entity extended Nader a helping hand after he appeared with his father on Al Kass TV, where he appealed for help to get the costly treatment.
SMA is a genetic disease that targets the central nervous system, hindering muscle movement. The father noted that the treatment is only available in the Gulf state’s Sidra Hospital and Switzerland.
“Now the only solution is an injection and in Qatar[…]Switzerland is far from us,” the father said.
Nader’s father told Al Kass that while his son was able to walk, he discovered the disease after seeing him struggle to get up at the age of one. He then took him to the American University of Beirut’s hospital, where he received a type 2 SMA diagnosis.
Those who are diagnosed with such a condition live between eight-to-10 years whereas those with type 1 of SMA, its rarest form, do not live beyond the age of three.
Al Kass TV host Khalid Jassim was then contacted by Qatar Charity to share a link for those willing to offer donations to secure the much-needed treatment for the child.
The genetic disease causes the loss of nerve cells in the spinal cord and is classified into four types that have varying impacts on those diagnosed. Once the muscles are unable to respond to the nerves’ signals, they can weaken and shrink.
The muscles that are most affected by SMA include the shoulders, hips, thighs, and upper back.
The treatment, Spinraza, was first approved by the United States Food and Drug Administration (FDA) in 2016. While it does not completely cure the disease, it can slow down or reverse SMA’s symptoms.
Then in 2019, the FDA approved Zolgensma, “the first gene-replacement therapy for a neuromuscular disease”, according to the Muscular Dystrophy Association.
In 2020, the FDA approved a third treatment, Evrysdi, for SMA in adults and children aged two months or older.
Globally, one in every 6,000 to 10,000 babies are born with SMA, with type 1 affecting more than half of all cases.