2 months ago
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Tenley has Type 1 Spinal Muscular Atrophy - the most severe form of the disease. Tragically, most children with Type I Spinal Muscular Atrophy (SMA1) don’t live past their 2nd birthday.
Nationwide Children’s is conducting the world’s first gene therapy clinical trial for SMA1, and we are optimistic about preliminary results. These results can be seen in patients like Tenley. She received an intermediate dose and has been able to meet significant milestones in her development since she was enrolled in the trial in 2014.
SMA1 patients are missing the SMN gene. The goal of the gene therapy trial is to replace the missing SMN gene with a healthy gene that’s injected into the bloodstream.