Is There Hope for Charlie Gard After U.S. Doctor’s Visit?

Amid the legal wrangling, these loving parents just want the best for their desperately ill son

The parents of baby Charlie Gard remain hopeful their 11-month-old son can get experimental treatment for a rare genetic condition despite the international legal battle they’ve been enduring.

On Monday, a professor of neurosurgery from the U.S. examined the baby — who is British — at Great Ormond Street Hospital in London. The child is unresponsive and on life support.

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Dr. Michio Hirano of Columbia University in New York City took a brain scan of Gard, who has rare mitochondrial DNA depletion syndrome, to determine whether the baby can receive experimental therapy. The baby’s parents — Chris Gard and Connie Yates — remain optimistic about their son’s situation.

The hospital in London believes the child’s condition is irreversible, however, and that the medical team should turn off life support. The baby’s parents want to get him to the U.S. for treatment. The Gard family has raised over $1.6 million and is ready to travel with the child, but the European court system has denied the parents the right to take their child elsewhere for care.

The hospital did give Hirano access to Gard’s medical records and an honorary contract, according to media reports. The honorary contract temporarily gives the doctor the same rights as one of the hospital’s own clinicians, as The Telegraph noted.

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Little Charlie has a condition characterized by “progressive muscle weakness and brain damage,” the BBC reported.

Hirano is developing a nucleoside bypass therapy. New York Presbyterian Hospital and Columbia University Irving Medical Center in the U.S. have agreed to admit and evaluate Gard.

Related: Baby Charlie Gets Support from President Trump

“Charlie’s condition is exceptionally rare, with catastrophic and irreversible brain damage,” the children’s hospital in London said in a statement on July 13. “Our doctors have explored every medical treatment, including experimental nucleoside therapies. Independent medical experts agreed with our clinical team that this treatment would be unjustified.”

Great Ormond Street Hospital has said it does not want the baby to suffer.

The struggle over Charlie Gard’s life continues — and many around the world are watching.

Hirano’s nucleoside treatment apparently has been used on “at least one American patient with a similar but less severe mitochondrial disease,” as STAT News reported. “In both cases, a mutation impairs the function of DNA in mitochondria, microscopic structures within cells that produce the energy they need to survive.”

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“Because the treatment is experimental, Columbia would need approval from the Food and Drug Administration to administer the compounds to Charlie under a ‘compassionate use’ exemption,” STAT News noted.

After Hirano finishes his examination of the child, he could make the request for FDA approval. The baby has a mutation in the RRM2B gene that codes for the production of an enzyme; in the short term, it keep cells functioning properly. A gene mutation, such as in Charlie Gard’s case, results in a dysfunctional enzyme.

STAT News reported that 18 patients worldwide have received nucleoside bypass therapy. The other patients have a mutation in a gene called TK2.

Doctors have never tried the therapy on anyone with baby Charlie’s specific form of the genetic mitochondrial depletion syndrome.

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