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在spinal muscular atrophy treatment這個產品中，有2篇Facebook貼文，粉絲數超過0的網紅，也在其Facebook貼文中提到， I hope this post gets as many eyes on this as possible - please take a few minutes to read if you can. Devdan is born with Type 2 Spinal Muscular At...

spinal muscular atrophy treatment 在 Facebook 的最佳貼文

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2021-08-08 12:58:25 有 32 人按讚
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I hope this post gets as many eyes on this as possible - please take a few minutes to read if you can.

Devdan is born with Type 2 Spinal Muscular Atrophy (SMA) – a rare condition that damages nerve cells in the brain and spinal cord. This leads to progressive muscular weakness and trouble with activities such as breathing, speaking, swallowing, and walking.

Zolgensma, is a ONE-TIME-ONLY gene therapy treatment for children aged under 2 years with SMA which costs $2.8 MILLION.

Devdan turns 2 in Oct so this is a race against time to raise this money before FDA will send this drug out. I myself have donated, and so far we have clocked over $1 million which is fantastic but we still have yet to reach our goal. Appreciate it if you can spare a donation - no amount is too small. 🙏🏼

Stay strong Devdan, always remember God is fighting with and for you. ❤️

DONATE HERE: https://rayofhope.sg/campaign/please-give-devdan-a-second-chance-of-life/

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spinal muscular atrophy treatment 在 元毓 Facebook 的最佳解答

元毓 By 元毓

2017-09-28 10:31:31 有 11 人按讚
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worth of notice:

quote:
"THE GOAL OF REPLACEMENT gene therapy is to replace faulty genes with normal ones, in the hope of producing significant benefits or even cures. The process involves packaging healthy genes into neutered viruses, usually relatively harmless adeno-associated viruses, which then act as vectors delivered in a single dose either into the bloodstream or the area where the disease is manifest. The neutered viruses are designed to attach to targeted cells and deliver the new genes into the nucleus of the cells, along with so-called promoters that help prompt the new genes to direct cells to make critical proteins that are otherwise lacking or ineffective. ...

...A big issue for the industry and investors will be gene therapy pricing. At a time when treatments for rare diseases that need to be dosed indefinitely can cost $400,000 annually or more, how should a potential one-time cure be priced? Gene therapy companies haven’t said yet how they plan to price treatments, and Wall Street analysts and investors have said price tags of anywhere from $1 million to $4 million per patient for serious diseases could be justified when there are no alternatives—or when less effective alternative treatments cost more over time....

The current treatment for spinal muscular atrophy, Biogen’s (BIIB) Spinraza, was approved by the FDA last year. It costs $750,000 for the first year’s treatment and $375,000 a year thereafter..... A pay-for-performance model might develop, with annual payments based on continued success, but that raises issues of portability....

...Replacement gene therapy reduces the chances of a potentially harmful off-target genetic insertion that could lead to cancer. But there are risks, including a possible immune-system response to the vectors. One of the most serious incidents occurred in 1999, when an 18-year-old patient receiving experimental replacement gene therapy died from a severe immune response....

...If these treatments ultimately stop working, it might be difficult to re-treat patients with the same therapy because their bodies probably will have developed immunity to the viral vectors that initially delivered new genes. That means the viruses probably would be attacked by the patient’s immune system. Some companies are working on ways to address this issue, such as using different viral vectors...."

https://goo.gl/sP8B8v

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What is spinal muscular atrophy (SMA)? SMA's an autosomal recessive disorder that causes alpha motor neurons in the spinal cord to die, ... ... <看更多>

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#20. Spinal Muscular Atrophy (SMA) | Johns Hopkins Medicine

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