Rare Disease Report

U.S. Doctor Estimates 10% Chance Unapproved Treatment Could Help Charlie Gard

JULY 13, 2017
James Radke
The BBC is reporting that during the afternoon session of the hearing involving Charlie Gard’s future, a U.S. clinician, whose name will not be released per legalities, spoke via video conference, and said that treating the 11-month old with an experimental nucleoside therapy has an estimated 10% chance of improving the child’s condition.
The American doctor has not personally seen Gard in person, but he said that based on what he has seen of the tests provided him, the boy’s brain shows "disorganisation of brain activity and not major structural brain damage".
The experiment procedure involving nucleoside treatment has been performed on patients with similar, but not identical, patients. He acknowledged that while it would be desirable to conduct further testing on rodents, that could take a minimum of 6 months to 2 years.
This is the first time a doctor with hands-on experience with the proposed treatment has spoken about the procedure.
The court hearing today was a special session scheduled before Judge Nicholas Francis to hear any new evidence to change the court’s earlier decision to not permit the parents to take the child out of England’s Great Ormond Street Hospital. There, it was determined by the treating doctors that Gard would not benefit from the experimental treatment. Instead, he should be taken off life support to put a stop to any pain he might be experiencing.
The case has captured the attention of many in the rare disease community, as well as world leaders, like Pope Francis and President Donald Trump.
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