Sharon Hesterlee's Blog – September 2013 Archive (2)

DIA Oligonucleotide Conference, Neuromuscular Session Summary

At a Washington meeting last week on oligonucleotide-based therapeutics  co-sponsored by the Drug Information Association (DIA) and the Food and Drug Administration, several speakers addressed recent developments in the use of antisense oligonucleotides for Duchenne muscular dystrophy.  Allison Durham of FaegreBD captured the highlights of those talks for PPMD.

Update on Sarepta’s AVI-4658 in Exon 51 Skipping Amenable Duchenne Muscular Dystrophy…

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Added by Sharon Hesterlee on September 27, 2013 at 2:26pm — 1 Comment

GSK and Prosensa announce primary endpoint not met in Phase III study of drisapersen

UPDATE AS OF 10/4/13: We received the following Q&A from GSK today, addressing many of the community's questions. Click here.

Today we learned that GSK’s phase III study of the exon 51 skipping drug drisapersen failed to show a statistically significant improvement in the six-minute walk test compared to placebo (…

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Added by Sharon Hesterlee on September 20, 2013 at 11:00am — 11 Comments

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