Eleven-year-old Marley Mansour loves baseball. She\u2019s in a kids league, and for every game she has a signature outfit \u2014 a different colored tutu every time she plays.\u202f<\/p>\n
She also loves bubbles and going to church. Her favorite food isn\u2019t pizza; it\u2019s “pizza pie with pepperoni.”
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When Marley was a baby her parents noticed she wasn\u2019t putting on weight, and walking was a struggle. She fell a lot and had several seizures. Marley also would throw up almost every meal.<\/p>\n
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A photograph of Marley Mansour as a baby is displayed in her family\u2019s living room in Rowlett on Thursday, Feb. 27, 2025.<\/p>\n
\u201cWe saw so many doctors, we saw so many specialists, and there was never any answer,\u201d her mother, Kayla Mansour, said. \u201cIt definitely was a very lonely path, to be honest.\u201d<\/p>\n
Finally, when Marley was 9, they got an answer: Genetic testing showed she was born with a mutation in a gene called NARS1. It\u2019s involved in making a protein that\u2019s critical for the central nervous system \u2013 but it wasn\u2019t identified until 2020.<\/p>\n
\u201cThere wasn’t a road map. I didn’t know the resources yet. I didn’t know the places to look,\u201d Kayla Mansour said. \u201cIt was quite the journey, waiting nine and a half years to all of a sudden having a name for it.\u201d<\/p>\n
A NARS1 mutation can lead to developmental delays, like with speech. Those affected may have seizures, intellectual disabilities, and problems with their nerves. Marley is one of about 100 people in the world known to have the condition.<\/p>\n
The day Marley finally got her diagnosis, doctors told her mom there wasn\u2019t a cure.\u202f She was already in physical, occupational and speech therapy as well as swimming. Marley saw a neurologist and tried anything her family had access to.<\/p>\n
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Kayla Mansour, left, guides her daughter Marley\u2019s hand as they draw a heart with chalk outside their home in Rowlett on Thursday, Feb. 27, 2025.<\/p>\n
But Kayla Mansour was determined to find anything. That\u2019s when she discovered a\u202fnonprofit that creates individual treatments for rare\u202fdiseases.<\/p>\n
Later this year, Marley may become the first person in the world to receive\u202fpersonalized therapy for her disease.
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\u201cIt modifies the NARS1 gene to help her to be able to make that protein\u202fproducts when previously she was not,\u201d said Dr. Kaitlin Batley, Marley\u2019s doctor. She’s a pediatric neuromuscular neurologist and director of the Pediatric Neuromuscular program at UT Southwestern and Children’s Health in Dallas.<\/p>\n
The therapy was developed for a variety of diseases but has never been used to treat Marley\u2019s particular genetic mutation.\u202f\u202fBatley thinks this is a step forward for the medical community.\u202f
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\u201cIt is something that also shows kind of the personalized medicine and\u202f direction for personalized medicine for the future, that we may be able to\u202f\u202fcreate therapies for diseases that previously had none,\u201d said Batley.
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Marley will need an injection in her spine every few months for the rest of her life. The hope is to slow the progression of her disorder. Her mom acknowledges there are a lot of unknowns with the treatment \u2013 including whether it\u2019ll even work.<\/p>\n
\u201cI just have a lot of gratitude, but I also am nervous as well. I’ve never even given this child an antibiotic. And here I am, you know, going to infuse an experimental medicine into her spine,\u201d Kayla Mansour said. \u201cWe don’t know what we’ll get out of the treatment, but we are just so hopeful that it’s going to improve her quality of life at a minimum.\u201d<\/p>\n
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Marley Mansour, 11, smiles at her parents while sitting on her bed on Thursday, Feb. 27, 2025, in Rowlett. At age 9, Mansour was diagnosed with a rare genetic disorder called NARS1. She will be the first patient to receive an experimental antisense oligonucleotide gene therapy to treat NARS1 symptoms.<\/p>\n
Despite fears, Marley’s dad, Jonathan Mansour, said he\u2019s happy his daughter will pioneer the treatment for other people.<\/p>\n
\u201cWe’re excited about it,\u201d he said. \u201cA little bit nervous, but for the greater goal, I think it’s a good thing for us and a lot of other kids.\u201d\u202f<\/p>\n
The medication Marley will receive is free, but the administrative costs aren\u2019t.\u202f The family has a support registry<\/a> in Marley\u2019s name going up to $100,000.\u202f<\/p>\n \u202f Marley\u2019s the oldest of three children. Her parents haven\u2019t told any of their kids \u2013 including Marley \u2014 about her condition. Jonathan and Kayla Mansour want to focus on them being children.<\/p>\n They hope one day, Marley is able to understand everything she overcame.<\/p>\n Olla Mokhtar is KERA\u2019s news intern. Got a tip? Email Olla at omokhtar@kera.org<\/a>.<\/p>\n
\u201cInsurance is not going to pitch in because it’s experimental for a couple of\u202fyears. All these things that we’re having to navigate and now a six-figure\u202f\u202famount to make sure our daughter can live a better life,\u201d Kayla Mansour said. \u201cWe don’t know how\u202fwe’re going to do it, but we’re sure going to try to figure it out.\u201d
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