April 10 - 13, 2013
Cold Spring Harbor Laboratory (CSHL)
Conference homepage link
Ryszard Kole, Sarepta Therapeutics
Adrian Krainer, Cold Spring Harbor Laboratory
Art Krieg, RaNA Therapeutics
Bruce Sullenger, Duke University
There was a great deal of discussion around this conference, and particularly the data presentation by GSK/Prosensa for their Duchenne Muscular Dystrophy DMD drug drisapersen. This drug is of course the key competitor to eteplirsen from Sarepta Therapeutics SRPT. Key links and info are archived in this post.
Cirak, S."Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy" (Talk)
Fiorillo, A.A. "Dystrophin-targeting microRNAs modify both Becker muscular dystrophy severity and exon skipping success" (Talk)
Kole, R. "Update on clinical trial of eteplirsen for treatment of Duchenne muscular dystrophy" (Talk)
Kraus, J.E. "Drisapersen treatment for Duchenne muscular dystrophy—Results of a randomized, double-blind, placebo-controlled clinical trial" (Talk)
Click for slides from the Prosensa drisapersen DMD presentation.