Stanford patient is first infant to receive lifesaving drug
N 2013, Zoe Harting became the first baby in the world to receive an experimental drug that her doctors hoped would save the lives of thousands of infants like her.

Zoe has spinal muscular atrophy type 1, a degenerative neuromuscular disease that kills most patients by their second birthday.

Before she began receiving the drug, 7-month-old Zoe was quite weak. She couldn’t sit up or roll over. She couldn’t move her legs at all, or lift her arms when she was lying down. She struggled to swallow. Her parents, John and Eliza Harting, knew that without an effective treatment, she would soon struggle to breathe. But no treatment had ever succeeded against SMA-1. So when the Hartings got a call from Stanford pediatric neurologist John Day, MD, PhD, asking if they would consider enrolling Zoe in a phase-2 clinical trial of an experimental drug called nusinersen, they agreed....

http://med.stanford.edu/news/all-news/2016/12/stanford-patient-is-first-infant-to-receive-lifesaving-drug.html
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