Another publication of a successful clinical trial here. This one is only mice and apparently large mammal trials are always a better indication of moving closer to human trials and actual therapy.
I never know whether to get excited about these kinds of reports or not. On the one hand, a treatment will be possible one day and will be a wonderful thing for all those patients and families that it will benefit. But I always read the part about it having success only in young animals and believe that treatment will always come too late to benefit Charlotte. Then I get depressed again. It is sometimes better to have no hope than false hope.
On the other hand, as this kind of therapy becomes more widely used in the future, they may be able to make smaller and smaller gains with less risk. This might mean that even though she wouldn't have normal vision, she might be able to have a slight improvement which would still be worth it.
I really should go and sort out getting genetic testing for her. We've been meaning to for ages and we want to be ready to hit the button should anything really become available.
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Hello. I agree with you. I have achromatopsia, and I am maybe too old to recover a normal vision, but I might have a small improvement. The fact that you found this scientific article just 4 days after its publication is striking : congratulations ! (and sorry for the bad english, I am french...)
ReplyDeleteBonjour, thanks but I am certainly no research genius. I cheat: Google Alerts!
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