New York Hospital Offers to Treat British Baby With Rare Disease – New York Times

Alternately, officials said they would be willing to ship an experimental drug to the Great Ormond Street Hospital in London, where the baby is now being treated, if the Food and Drug Administration approves.

American physicians would advise their medical staff on administering it if they are willing to do so, the statement said.

Charlie Gard was diagnosed with an extremely rare form of a disease called mitochondrial DNA depletion syndrome, believed to affect just over a dozen children worldwide. The syndrome prevents cells from producing the energy needed to sustain organs.

The baby was brought to the London hospital on Oct. 11, when his parents, Connie Yates and Chris Gard, both in their 30s, noticed he was not growing and could not lift his head. He has been there since, breathing with the help of a ventilator and fed through a tube.

He is deaf and suffers from persistent seizures, and appears to have suffered brain damage.

Researchers at Columbia University have provided an experimental treatment to a child in Baltimore, Art Estopinan Jr., suffering from a similar but less severe form of the syndrome.

The childs father, Art Estopinan, said that he was contacted by Ms. Yates and Mr. Gard, and in turn asked the researchers at Columbia University if they could help Charlie Gard, as well.

Art Jr. was 18 months old in 2012 when doctors diagnosed a form of mitochondrial DNA depletion syndrome, and said he had less than two months to live.

Everyone told me the same thing: There is no medication, there is no cure, Mr. Estopinan said.

The treatment, called nucleoside therapy, is not approved by the F.D.A., but can be requested under exceptions for compassionate use.

Mr. Estopinan said with treatment, his son has slowly but steadily become stronger. Now 6, Art Jr. cant walk, but he can move his hands and feet.

He breathes with the help of a ventilator, is fed through a tube and needs round-the-clock care.

Mr. Estopinan said he was speaking out because my wife and I believe that little Charlie Gard should be given a chance, because we believe there is hope.

Ms. Yates and Mr. Gard have raised about $1.7 million to pay for the experimental treatment and to travel to the United States for care.

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New York Hospital Offers to Treat British Baby With Rare Disease - New York Times