Understanding the Experimental Treatment For Baby Charlie Gard and How It May Work
등록일 2019년 01월 07일 목요일
수정일 2017년 07월 20일 목요일

The world has turned its attention in towards young Charlie Gard, a baby in the United Kingdom with a potentially deadly mitochondrial disease.His parents continue to look to provide him with treatment in the United States but in spite of their efforts UK doctors insist that he be left to die.

There is a good chance for Gard to be treated for mitochondrial depletion syndrome, a condition that causes muscle weakness and organ failure.A particular medicine could be utilized as a means of helping to restore how well his mitochondrial DNA functions, thus potentially reversing the condition that puts his body at risk.

The medicine has been considered as a possible choice in helping Gard recover and develop his body again.However, there is only one recorded human test that was performed but has shown strong promise in terms of allowing the body to recover.

Understanding the Condition

Mitochondrial depletion syndrome has been found to keep the DNA that the cells require from forming.As this occurs, the cells will weaken and cause muscles and brain functions to wear out.This in turn keeps a person from being able to breathe on one's own or to have any kind of control over one's body.

A treatment being utilized at Columbia University may help to produce mitochondrial DNA.This is needed to help produce energy that can be used by cells.These are required to allow muscles to function normally, thus allowing critical organs around the body to operate as usual.

Critical nucleosides needed to keep the body active and functional have to be analyzed as well.There is the potential that such nucleosides could be restored within the body to potentially reverse the problem.This point has been the influencing factor to figuring out what can happen as a means of treating difficult problems within the body's cells.

How the Treatment Works

An experimental treatment to control mitochondrial depletion syndrome has been developed at Columbia University.The treatment is designed as an oral medicine product that administers the correct nucleoside that the body needs in order to improve upon how well its mitochondrial DNA can function.

The medication has been tested on mice that have shown some improvement upon using the medicine.It has been extremely difficult to try and figure out if this is suitable for use among people although one person has successfully used it in the past.

Five years earlier, a child who was a year old had utilized the medicine at Columbia.Art and Olga Estopinan took their child Art Jr.to Columbia for the treatment.Young Art had a condition similar to Gard's and was considered to be terminal.However, the condition that the child had is not as intense as what Gard is experiencing.

The treatment helped Art to restore some motor functions and brain activities.He is currently six years of age and has been learning a few words.While he requires a motorized wheelchair and a feeding tube while getting three to four medical treatments each day, he has shown some improvements over time.The medicine has helped Art to slowly restore normal functions in his body although it is uncertain as to how long it would take for him to receive full control over his body.

The Estopinan family is encouraging the Gard family to come to Columbia to receive this treatment.This is believed to be the best potential for Gard to receive a chance to live and to restore body functions.The potential for the treatment to work is uncertain though due to how Gard's situation is much more serious than what most others with the condition have.

Will Charlie Travel?

The potential for Charlie Gard to receive this treatment is strong in that his family has received the funding to get him out to Columbia.A ruling from the UK High Court has still blocked this, citing a need to review evidence with regards to how his condition could be treated.

It is uncertain as to whether he will travel to Columbia for the proper treatment that he could receive.A further decision to possibly allow him to travel to the United States is expected to be made before the end of July.He has been given legal permanent residence in the United States by Congress as a means of allowing him to get into the country to receive the treatment that he would require.

The situation will continue to be observed by people around the United States and United Kingdom.This especially comes amid concerns over healthcare and the desire to help people who are struggling with particular medical conditions, particularly ones that are not as commonplace or noticeable.

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