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Man with rare ALS improves after first-ever RNA therapy targeting genetic mutation

Results published; researchers call findings exciting but cautious

1 Sep 17 · 6d ago · 1 article · 2 posts · 3 sources · development 1 of 1

The case study was published in a peer-reviewed journal this week. Neurologist Steve Vucic called the results an "exciting first step" while noting it is too early to know if the treatment stops disease progression or cures the condition. Fleur Garton noted that similar antisense therapies could be developed for people with common, rare, or multiple genetic mutations driving ALS.

Results published; researchers call findings exciting but cautious
nature.com

“the results are an "exciting first step", although it is too early to know whether the treatment could stop disease progression or be a cure”

Steve Vucic

Trial participant First patient treated with mutation-targeting RNA therapy for ALSSteve Vucic Neurologist and ALS researcher, University of SydneyFleur Garton Neurological disease researcher, University of Queensland

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Peak 2 pieces in two hours at Sep 17, 7 PM; 6 pieces over 6 days (2 articles · 4 posts) Sep 17, 7 PM — 2 pieces · 1 article · 1 post — Mastodon 1, Newswires 1Sep 17, 9 PM — quietSep 17, 11 PM — quietSep 18, 1 AM — quietSep 18, 3 AM — quietSep 18, 5 AM — quietSep 18, 7 AM — quietSep 18, 9 AM — 1 piece · 1 article — Google News 1Sep 18, 11 AM — quietSep 18, 1 PM — quietSep 18, 3 PM — quietSep 18, 5 PM — quietSep 18, 7 PM — quietSep 18, 9 PM — quietSep 18, 11 PM — quietSep 19, 1 AM — quietSep 19, 3 AM — 1 piece · 1 post — Bluesky 1Sep 19, 5 AM — quietSep 19, 7 AM — quietSep 19, 9 AM — 1 piece · 1 post — Reddit 1Sep 19, 11 AM — quietSep 19, 1 PM — quietSep 19, 3 PM — quietSep 19, 5 PM — quietSep 19, 7 PM — quietSep 19, 9 PM — quietSep 19, 11 PM — quietSep 20, 1 AM — quietSep 20, 3 AM — quietSep 20, 5 AM — quietSep 20, 7 AM — quietSep 20, 9 AM — quietSep 20, 11 AM — 1 piece · 1 post — Reddit 1Sep 20, 1 PM — quietSep 20, 3 PM — quietSep 20, 5 PM — quietSep 20, 7 PM — quietSep 20, 9 PM — quietSep 20, 11 PM — quietSep 21, 1 AM — quietSep 21, 3 AM — quietSep 21, 5 AM — quietSep 21, 7 AM — quietSep 21, 9 AM — quietSep 21, 11 AM — quietSep 21, 1 PM — quietSep 21, 3 PM — quietSep 21, 5 PM — quietSep 21, 7 PM — quietSep 21, 9 PM — quietSep 21, 11 PM — quietSep 22, 1 AM — quietSep 22, 3 AM — quietSep 22, 5 AM — quietSep 22, 7 AM — quietSep 22, 9 AM — quietSep 22, 11 AM — quietSep 22, 1 PM — quietSep 22, 3 PM — quietSep 22, 5 PM — quietSep 22, 7 PM — quietSep 22, 9 PM — quietSep 22, 11 PM — quietYesterday, 1 AM — quietYesterday, 3 AM — quietYesterday, 5 AM — quietYesterday, 7 AM — quietYesterday, 9 AM — quietYesterday, 11 AM — quietYesterday, 1 PM — quietYesterday, 3 PM — quietYesterday, 5 PM — quietYesterday, 7 PM — quietYesterday, 9 PM — quietYesterday, 11 PM — quietToday, 1 AM — quiet 1
1Results published; researchers call…
Sep 18Sep 19Sep 20Sep 21Sep 22yesterdaynow · 3:51 AM ET

Reported in the same hours no headline names this development itself — these 1 claim were published in its stretch

  1. 2 outlets First for RNA therapy: man with rare motor-neuron disease improves after treatment

    first by Nature, 5d ago · also Nature News

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