An 11-year-old Rowlett girl is set to become the first person in the world to receive treatment for NARS1, a rare genetic disorder, the Dallas Morning News reports.
Why it matters: The California-based nonprofit that developed the treatment for Marley Mansour hopes it can help other patients with similar genetic profiles.
Stunning stat: Fewer than 100 people globally have been identified with NARS1, the DMN reports.
How it works: The disorder affects the body's protein production, causing developmental and neurological challenges.
Marley was diagnosed in early 2023 and cannot speak.
How it works: The nonprofit n-Lorem develops individualized antisense oligonucleotide therapy treatments — known as ASO — for patients with ultra-rare conditions.
Marley's treatment was designed to target the defective RNA in her body and improve her communication difficulties and peripheral neuropathy.
A UT Southwestern Medical Center doctor will administer the treatment via a spinal tap, per the DMN.
Follow the money: n-Lorem will pay for the treatment, which costs about $1.2 million, but Marley's family will pay the other related expenses.
/2025/05/05/1746475978795.gif?w=3840)