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Huntington's disease breakthrough 'is like winning the lottery 10 times over' (www.bbc.com)

submitted 1 day ago by MicroWave@lemmy.world to c/health@lemmy.world

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Until now there has been no treatment. But on Wednesday researchers reported how a new gene therapy had managed to slow progress of the disease by 75%.

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[–] pulsey@feddit.org 6 points 1 day ago* (last edited 1 day ago) (1 children)

So far, just the headline findings have been released. Scientists say the full study needs to be published and assessed by independent experts to properly assess what has been achieved.

The study is also relatively small - there were just 29 participants who were followed for 36 months. This is not unusual for gene therapies, and others have made it onto the NHS off the back of small-scale trials. The case for this treatment is particularly strong given there are no current treatment options for the disease.

But even if this therapy is licensed, it is only going to help a small portion of Huntington's disease patients as it's aimed at those with early-stage symptoms or those who haven't developed any yet. The complex nature of the surgery also means only specialist centres will be able to perform it.

Some scientists have pointed out that it is not yet certain the benefits will last long-term.

[–] atro_city@fedia.io 1 points 21 hours ago

It's still amazing progress. First this, then they'll understand more of why it works and why it doesn't. That will allow them tailor it or find other approaches to have the same or better results.

Progress is slow.