NEW YORK—A leading academic medical centre in New York City has offered to treat Charlie Gard, an 11-month-old infant in Britain who was born with a rare and fatal genetic disease.European courts have ruled that he should be taken off life support, as there are no effective treatments for his condition. His parents were denied permission to bring him to the United States for experimental therapy.The court decisions captured the attention of Pope Francis and U.S. President Donald Trump, who tweeted Monday that if the United States could help, “we would be delighted to do so.”Read more: Donald Trump offers help to terminally ill British babyDespite big offer from Donald Trump, little has changed for critically ill British babyIn a statement issued Thursday by NewYork-Presbyterian Hospital/Columbia University Medical Center, officials agreed to admit Charlie as an in-patient as long as he could be transferred safely and there were no legal or regulatory barriers to treating him with an experimental medication.Alternately, officials said they would be willing to ship an experimental drug to the Great Ormond Street Hospital in London, where the baby is being treated, if the Food and Drug Administration approves.U.S. physicians would “advise their medical staff on administering it if they are willing to do so,” the statement said.Charlie 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 taken to the London hospital 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 Estopian Jr., suffering from a similar but less severe form of the syndrome.The child’s father, Art Estopinan, said that he had been contacted by Yates and Chris Gard and in turn had asked the researchers at Columbia University if they could help Charlie 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,” Estopinan said.The treatment, called nucleoside therapy, is not approved by the FDA but can be requested under exceptions for compassionate use.Estopinan said that, with treatment, his son had slowly but steadily become stronger. Now 6, Art Jr. can’t 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.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.”Yates and Gard have raised about $2.2 million to pay for the experimental treatment and to travel to the United States for care.
TorontoStar, July 07, 2017