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Courtesy of BBC Online:
Gene Therapy Trial for Duchenne Dystrophy to Begin in England
Duchenne is the most deadly known form of the 40-odd diseases lumped under the catch-all term "muscular dystrophy." Most of its victims die in boyhood, and not the least bit pleasantly.

This treatment is based on work funded by the Muscular Dystrophy Association here and elsewhere. Please remember this next time you get the urge to diss MDA or its national chairman in my presence.

Date: 2007-10-20 12:59 pm (UTC)
From: [identity profile] sodyera.livejournal.com
I used to be in regular contact with Jackie Nieves's brother Andrew, who has Duchennes. Unfortunately, I haven't heard anything about him lately. But from what I've caught, the new drug wouldn't help sufferers regain muscle control beyond a pretty young age. I do not deny this important forward step, but there's still so much more to do.

February 2023

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