A Fort Worth toddler was the first person in Texas to be treated with a new, high-dose drug for a rare genetic disorder.<\/p>\n
Jesse Gonzalez Vasquez, 2, was born with spinal muscular atrophy. The disease affects the body\u2019s motor neurons, which are the roots of your motor nerves in your spinal cord, said Dr. John Brandsema, a pediatric neurologist at the Children\u2019s Hospital of Philadelphia and an expert on spinal muscular atrophy, or SMA. SMA affects roughly 1 in 10,000 live births, according to the Muscular Dystrophy Association<\/a>.<\/p>\n Brandsema explained that humans are born with one pool of motor neurons for life, and SMA slowly robs individuals of their motor neurons over time, leaving them with difficulty walking or running, holding up their heads, or even swallowing food and liquid safely. The disease can be paralyzing, or even fatal.<\/p>\n SMA used to be the most common genetic cause of infant death, Brandsema said.<\/p>\n When Lupita Vasquez, Jesse\u2019s mom, learned her son\u2019s diagnosis, she was devastated.<\/p>\n \u201cThey can\u2019t really promise you that your baby\u2019s going to walk,\u201d Vasquez said.<\/p>\n But Jesse was born during a revolution in how patients with SMA are treated. In the last decade, a tranche of treatments for the disease has become available, making the disease that was once a devastating diagnosis a more manageable condition.<\/p>\n