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Who is Michio Hirano? Charlie Gard doctor from the US who believed nucleoside bypass therapy could help tot

DR MICHIO Hirano is the American neurologist who offered to provide revolutionary treatment to help British terminally ill tot Charlie Gard, who has now passed away.

New York-based Dr Hirano had been hoping to oversee a trial in which the 11-month-old, who has a rare genetic condition and brain damage, would undergo nucleoside bypass therapy.

 Dr Michio Hirano is the US doctor who wanted to give Charlie experimental treatment
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Dr Michio Hirano is the US doctor who wanted to give Charlie experimental treatment

Who is Dr Hirano?

Dr Michio Hirano is based at the Columbia University Medical Center where he is a professor of neurology.

He has been a doctor for more than 30 years having been awarded his Masters Degree from the Albert Einstein College of Medicine in the Bronx in 1986, after he had received his BA from Harvard College.

His mother Keiko taught Japanese at the Bronx High School of Science and his father was a professor of pathology and neuroscience at Albert Einstein, reported the .

On Columbia's it states that he is an elected member of the American Neurological Association and has been named among America's Best Doctors.

 Charlie's parents wanted their son to undergo an experimental treatment course
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Charlie's parents wanted their son to undergo an experimental treatment courseCredit: PA:Press Association

What is Dr Hirano offering to help Charlie?

Nucleoside bypass therapy is an experimental treatment for mitochondrial depletion syndrome.

Charlie suffered from a very rare form of mitochondrial depletion known as RRM2B.

This mutation affects the cells that are responsible for energy production.

In theory the experimental treatment, which is taken as an oral medication, could have seen Charlie’s mitochondrial DNA synthesise again by giving him the naturally occurring compounds his body isn’t able to produce.

 Chris Gard and Connie Yates held hands as they arrived at the High Court for a preliminary hearing on July 10
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Chris Gard and Connie Yates held hands as they arrived at the High Court for a preliminary hearing on July 10Credit: EPA

Has this experimental treatment worked on other sufferers?

A US expert said 18 people have been treated with nucleoside bypass therapy, but none were in a condition as severe as Charlie’s.

Art Estopinan's son, Art Jr, suffers another form of mitochondrial depletion called TK2 - a "cousin" disease to Charlie's.

But Charlie's condition was  more serious because his brain as well as his muscles are affected.

Five years ago, Art Jr appeared to be facing death when he became the first human to be given nucleoside medication, in a trial overseen by Dr Hirano.

Art said his son had a "new lease of life" by the drug and can now stand with the help of a brace and say a small number of words.

He told Sun Online: "We were elated. It was like God had answered our prayers."

Dr Hirano and his staff had secured permission from the Food and Drug Administration agency (FDA) in the States to test the therapy on Art Jr.

“It is not a cure, but it is a treatment,” Hirano told the . “It has modest benefits, and we’re working on ways to improve the therapy.”

 Art Estopinan Jr, now six, became the first person to have the therapy in 2012
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Art Estopinan Jr, now six, became the first person to have the therapy in 2012Credit: Art Estopinan

What has Dr Hirano said about Charlie's case?

Dr Hirano flew to the UK to meet the experts at Great Ormond Street who had been treating eleven-month-old Charlie.

He said that he believed the experimental therapy could boost the tot’s brain function, and revealed the treatment had up to a 50 per cent success rate on patients with a similar condition.

Speaking over video-link, Dr Hirano said he believed there was “at least a 10 per cent” chance that Charlie’s muscle strength could improve and the therapy was “worth trying”.

He also hinted that Charlie may not be permanently brain damaged – as argued by doctors at Great Ormond Street Hospital.

The neuroscientist believes new drugs have a “small but significant” chance of reversing brain damage caused by Charlie’s rare genetic condition.

On July 19, Dr Hirano failed to convince Great Ormond Street medics that his experimental treatment offers a lifeline to little Charlie.

He met with four consultants and Charlie’s mum Connie. 

Connie and Charlie's dad Chris Gard have withdrew an application at the High Court to send Charlie to the States for the treatment.

Does he have a 'financial interest' in Charlie's case?

Dr Hirano was forced to deny he was in it for the money and slammed reports he would have benefited financially from the therapies.

He said: "I became involved in Charlie’s case when I was contacted by his parents, and I subsequently agreed to speak with his doctors to discuss whether an experimental therapy being developed in my lab could provide meaningful clinical improvement in Charlie’s condition.

“As I disclosed in court on July 13, I have relinquished and have no financial interest in the treatment being developed for Charlie’s condition.

“Unfortunately, a MRI scan of Charlie’s muscle tissue conducted in the past week has revealed that it is very unlikely that he would benefit from this treatment.”

It comes after Great Ormond Street slammed the doctor and outlined their “disappointment" in him.

In an extensive statement, said: "When the hospital was informed that the Professor had new laboratory findings causing him to believe NBT (nucleoside bypass therapy) would be more beneficial to Charlie than he had previously opined, GOSH’s hope for Charlie and his parents was that optimism would be confirmed.

 Baby Charlie Gard was in the 'terminal stages' of the mitochondrial DNA depletion syndrome during the legal battle
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Baby Charlie Gard was in the 'terminal stages' of the mitochondrial DNA depletion syndrome during the legal battle

"It was, therefore, with increasing surprise and disappointment that the hospital listened to the Professor’s fresh evidence to the Court.

"On 13 July he stated that not only had he not visited the hospital to examine Charlie but in addition, he had not read Charlie’s contemporaneous medical records or viewed Charlie’s brain imaging or read all of the second opinions about Charlie’s condition (obtained from experts all of whom had taken the opportunity to examine him and consider his records) or even read the Judge’s decision made on 11 April.

"Further, GOSH was concerned to hear the Professor state, for the first time, whilst in the witness box, that he retains a financial interest in some of the NBT compounds he proposed prescribing for Charlie.

"Devastatingly, the information obtained since 13 July gives no cause for optimism.

"Rather, it confirms that whilst NBT may well assist others in the future, it cannot and could not have assisted Charlie."

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