November 19, 2018 at 4:13 pm #163989Reply To: Is crispr going to cure us all?
I thought Crispr was a protein that attacks an enzyme. prob wrong tho lol
I am fairly open minded and live in hope as the say. The Advancement in SMA is very encouraging. So hopefully that will birth some positive lab results for the rest of the MD family
"""""""What doesn't kill you makes you stronger""""""catatudeModeratorPosts: 1,002Joined: 20/09/2010December 3, 2018 at 11:25 pm #164711Reply To: Is crispr going to cure us all?
Do you not think?
When you read about it it all sounds positive, but I wouldn’t like to guess how long it would take.
Some dogs were cute from muscular dystrophy. https://www.google.co.uk/amp/s/www.bbc.co.uk/news/amp/health-45355556
Rats have also been cured.
How lucky are they
Some other muscular dystrophies are apparently in line to be tested too.
Probably be at least 10 years though? I’ll probably be dead by then hahaMded89ParticipantPosts: 28Joined: 10/03/2014December 4, 2018 at 12:51 pm #164725Reply To: Is crispr going to cure us all?
I too had read of a cure for FHSD some years ago but that has disappeared into the research woodwork again. Like many of these projects the research is time consuming and expensive and clearly MD does not see the same amount of research money put in as other deserving causes. Many of the money givers will look at cost benefit analysis of what they give. How many will it help, will it give quick results, what difference can it make etc. Even if a cure is proven at the animal level (which I do not agree with), there will be many years of testing in other ways before it can be tested on volunteers. Then of course it will have to go through various regulatory bodies, and of course NICE. The latter will determine if it would be funded by the NHS and will do so on cost benefit analysis alone in my opinion. If a treatment costs, say, £5000, will the NHS be able to save that amount and more by withdrawing other no longer needed treatments? If not it is unlikely to be funded. The pressure of numbers will have a significant input into the debate too. In the UK there are around 70000 people with the condition. If we all had the same variety such numbers would allow for bulk manufacture of the ‘cure’ and bring costs down. As you will no doubt know we are not all the same, and thus individual ‘cures’ would need to be found, and with some groups perhaps listing in the hundreds or less, that may well prove difficult and expensive.
Of course if everything gets through NICE you then still have to get the ‘cure’ from a specialist who will need to make a clinical judgement as to whether it is appropriate, and indeed whether you have that particular condition. A simple look through this forum will show how difficult it is to get a firm diagnosis, before any treatment occurs.
Thus, as you can see, the path is not only rocky, it is steeply uphill. With that in mind we should be grateful to the dedicated researchers who are treading this path for us. Be under no illusion though it will not happen quickly, unless a miracle occurs. I am retired now and I do not expect to see a cure for FHSD in my lifetime. I just hope it will be in time for my son.
SO many love songs, so little love.embayweatherModeratorPosts: 8Joined: 02/11/2015December 5, 2018 at 12:35 pm #164739Reply To: Is crispr going to cure us all?
Following on from this thread I came across this link today
It is telling us about worms, destined for the International Space Station. There they were to be used for work on treatments for Muscular Dystrophy. So things are still going on, especially in high (in this case very high) places.
SO many love songs, so little love.embayweatherModeratorPosts: 8Joined: 02/11/2015
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