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Two gene therapies for sickle cell disease approved in U.S.

11 Comments
By LAURA UNGAR

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This is just a small step forward since the therapy is still risky, complicated and extremely costly, but it signals progress in the field of gene therapy, year after year the medical interventions are more effective, less dangerous and a little bit less expensive. Seeing the advance in the last 5 years it makes people be very optimistic about what will be available in 5 or 10 years more.

2 ( +4 / -2 )

Wow, that is pretty big news. Basically the first attempt to cure a widespread genetic disease in adults.

5 ( +5 / -0 )

The list price for Bluebird Bio is $3.1 million and for Vertex, $2.2 million.

Had to read that twice...

Wouldn't it be better to screed potential parents and where possible, avoid diseases babies in the first place?

-6 ( +1 / -7 )

Wouldn't it be better to screed potential parents and where possible, avoid diseases babies in the first place?

Since this means selectively infringing in the reproductive rights of the population of an already discriminated ethnic group it is deeply unethical and immoral. The solution you propose is equivalent to eugenics, something that the social development of humanity already left behind.

Gene therapy is the opposite, because it not only provides a limited solution to the problem but because it also leads to further advancement that could in the future solve many other congenital problems without infringing in the fundamental rights of individuals.

3 ( +4 / -1 )

The solution you propose is equivalent to eugenics,

No, the solution I propose is common sense. If you are a carrier of the CS gene and know this then why on earth would any sane person have a kid with another carrier?

Or if they do, pay for their medical bills themselves.

That $3.2 million for one patient would pay for an awful lot of screening.

-5 ( +1 / -6 )

This is just a small step forward since the therapy is still risky, complicated and extremely costly, but it signals progress in the field of gene therapy, year after year the medical interventions are more effective, less dangerous and a little bit less expensive. Seeing the advance in the last 5 years it makes people be very optimistic about what will be available in 5 or 10 years more.

-4 ( +1 / -5 )

Genetic disorders

Albinism. Albinism is a group of genetic conditions. ...

Angelman syndrome. A rare syndrome causing physical and intellectual disability. ...

Ankylosing spondylitis. ...

Apert syndrome. ...

Charcot-Marie-Tooth disease. ...

Congenital adrenal hyperplasia. ...

Cystic fibrosis (CF) ...

Down syndrome.

FragileX syndrome.

Klinefelter syndrome.

Triple-X syndrome.

Turner syndrome.

Trisomy 18.

Trisomy 13.

and more

0 ( +1 / -1 )

The therapies will give hope to the suffering.

1 ( +1 / -0 )

This is just a small step forward since the therapy is still risky, complicated and extremely costly, but it signals progress in the field of gene therapy, year after year the medical interventions are more effective, less dangerous and a little bit less expensive. Seeing the advance in the last 5 years it makes people be very optimistic about what will be available in 5 or 10 years more.

Any point on just copy-pasting the first comment I made?

0 ( +0 / -0 )

No, the solution I propose is common sense.

That is definitely not the case, is ethically unacceptable and no medical professional supports it. The actually ethical measure is to support the people with the genetic predisposition so they can have productive lives thanks to the support of society, this is the same process that have helped make productive members of society people that suffered from other genetic problems like hemophilia.

Between people that search to limit the reproductive freedom of people based on racial profiling and those that search for effective treatments to solve the problem definitely the latter are in the ethical side of the issue.

As jacknbox commented, this is as unethical as doing the same for any other disease with genetic predisposition.

-3 ( +0 / -3 )

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