In 2017, Farah Mirizadeh was diagnosed with Spinal Muscular Atrophy (SMA) and given maybe seven to eight months to live, according to her parents.
Today, 6-year-old Farah is alive, talking, and moving despite the disease.
Her father, Aykhan Mirizadeh, and mother, Sabina Barkhu, spoke to KIRO 7 about their experience raising a child with SMA.
“We discovered that she had SMA when she was five months old because that’s when she stopped eating.”
Barkhu has characterized their home as a miniature Intensive Care Unit with all kinds of devices meant to help Farah.
“It’s been a whirlwind … we take it one day at a time.”
Her parents say that Farah came home with them after she was born and there was no indication she had any concerns; she seemed a perfectly normal baby.
Researchers at Johns Hopkins University say that SMA may not present itself as a condition until a little later in life.
Farah was only a few months into her life when doctors learned she had SMA - a severe, progressive neuromuscular disease.
According to the Centers for Disease Control: “Spinal Muscular Atrophy is a genetic disorder that affects approximately 1 out of every 10,000 people. Most cases of SMA occur when a segment of a gene called SMN1 is missing, resulting in the gene being unable to make protein. SMN1 is primarily responsible for making survival motor neuron (SMN) protein, which is required for maintaining normal, motor neuron function. Without enough SMN protein, motor neurons will degenerate, causing progressive loss of controlled muscle movement. There are different levels of SMA severity. Typically, the earlier the onset of the disease, the more severe the symptoms. SMA can affect a baby’s ability to swallow, breathe, sit, and walk. Without early treatment, SMA can also result in premature death.”
Farah’s parents say she is taking the first-ever at-home treatment for SMA called Evrysdi, which was only just approved for use in children in August 2020, according to the FDA.
Both Sabina and Aykhan learned that SMA does not impact the ability to think and learn.
Aykhan says the medication has manifested everything she’s noticed for years.
“SMA kids... they can’t move so, all they do is collect info. She’s sitting. She just started talking this summer. We’ve been waiting to hear her for five years.”
Sabina is just glad that the initial prognosis that Farah would only have a few months to live has improved with the medication.
“She’s still with us, she’s thriving wonderfully. There is hope. You just have to find the right people, the right doctors, the right support system, and hold on to that glimmer of hope because we did.”
Her parents did not screen for the disease before she was born, so now they are working to raise awareness on the issue.
They said that in 2020 Washington joined a list of states screening all newborns for SMA.
That screening process could allow parents to use one of two injectable treatments in newborns approved by the FDA; those treatments essentially adjust gene behavior or functions or repair genes that cause SMA. The sooner you start the treatment, the better the outcome, according to researchers.
The company that developed the medicine Farah is using, Genentech, said last year that it has received approval for the medicine to be used in more than 90 countries and is now being used to treat more than 8,000 patients worldwide.
In 2020, the company issued a news release that gave details about the medical breakthrough.
“Given the majority of people with SMA in the U.S. remain untreated, we believe Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease,” said Levi Garraway, chief medical officer and head of Global Product Development at Genentech. “The strength and resolve of the SMA community has continually inspired us as we developed this first-of-its-kind medicine for SMA, so today we celebrate our collective accomplishment together with them.”
“Throughout their lives, many people with SMA may lose their ability to perform critical movements, which can impact the ability to independently participate in aspects of daily life and even be life-altering,” said Kenneth Hobby, president of Cure SMA. “The approval of Evrysdi is an eagerly awaited milestone for our community. We appreciate Genentech’s commitment to reflecting the full scope of the real-world SMA population in their clinical trial program and developing a treatment that can be administered at home.”
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