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Regenerative Medicine and the Right to Try

Published onAug 07, 2022
Regenerative Medicine and the Right to Try

18 Wake Forest J. Bus. & Intell. Prop. L. 590

Charlie Gard appeared healthy at the time of his birth on August 4,
2016. Two months later, he was hospitalized in intensive care at Great
Ormond Street Hospital (GOSH) in London, where he was diagnosed
with infantile onset encephalomyopathic mitochondrial DNA depletion
syndrome (MDDS), which was caused by a mutation in his RRM2B
gene. MDDS is typically diagnosed during a child’s infancy, and
Charlie’s condition worsened rapidly. He became paralyzed and
dependent on respiratory support and suffered increasing damage to his
brain. Charlie’s mother began to do research online about his
condition, and soon his parents sought to take him to the United States
to pursue an experimental nucleoside treatment, which had apparently
produced some benefit in MDDS infants with a mutation in a different
gene—the TK2 gene. Through a GoFundMe campaign, Charlie’s
parents raised sufficient funds to travel to the United States.

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