Gene Therapy for SMA Type 1: Evelyn's Story

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"Something like this has never been achieved before." - Jerry Mendell, MD

Spinal Muscular Atrophy or SMA Type 1 is the leading genetic cause of death for infants today. A devastating fact the Villarreal family knows all too well. Their first daughter, Josephine, was diagnosed with SMA Type 1 and passed away at 15 months. 95 percent of babies born with the disease die before their second birthday. SMA rapidly robs babies of their ability to move, talk, swallow and eventually breathe. When Milan and his wife Elena found out they were pregnant with their second child, Evelyn, they prayed for a healthy baby. Unfortunately, that was not the case. Evelyn tested positive for SMA Type 1. Soon after, Evelyn’s family found out about the clinical trial going on at Nationwide Children’s Hospital. “It’s the first time we’ve been able to apply gene therapy to any neuromuscular disease,” said Dr. Jerry Mendell, principal investigator with the Center for Gene Therapy. Evelyn was given the gene therapy and it worked. “We start seeing changes as early as two months after treatment,” said Mendell. Today, Evelyn is a healthy, normal child. Something we never have been able to say about a child with SMA Type 1. Now, her parents are thinking long-term and planning for college.
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