Duchanne muscular dystrophy is much more common than Rett (incidence 1:10,000 for Rett Vs 1:3500 for DMD)Sarepta drug for DMD in phase 2 trial included 12 patients 4 in three arms is expected to apply for fda approval based on these results in early 2014.Serepta current market cap is $USD 1.5
Billion (they only have DMD drug and eventual expected approval priced into MC, other anti viral products in very early developmental stage)
So all of us can imagine what may happen on positive Rett trial result and near the time for fda filling, general understanding is that positive Rett trial will open the door for market interest in seeing a positive outcome in other indications
Apology if this is already discussed I have only started to follow this company
http://www.thestreet.com/story/11891228/1/hey-sarepta-investors-glaxos-drisapersen-data-revealed-early.html?puc=yahoo&cm_ven=YAHOO
http://seekingalpha.com/article/1334611-muscular-dystrophy-drug-succeeds-sarepta-buyers-flood-in
http://www.sareptatherapeutics.com/our-programs/rare-diseases/duchenne-muscular-dystrophy/
rett vs dmd
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