PS - check the Twitter folks who provided these links, all are key follows for those interested in this area.
PPS - as always, inclusion of a link here does not imply I endorse or agree with the views of the authors
DMD mother and advocate Jenn McNary speaks at the Forbes Healthcare Summit
Why I'm More Confident in Sarepta Than Ever Before
Dan Rosenblum (The Street) - aka @sharkbiotech
THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study
McDonald et al. Muscle & Nerve, September 2013 (h/t @themicrokid)
World Muscle Society Congress: Next Generation Exon-Skipping & Clinical Trial Updates
Sharon Hesterlee (Parent Project Muscular Dystrophy) (h/t @andybiotech)
Slides from September 27, 2013 Drisapersen data presentation by Prosensa
(h/t @sport234a)
DIA Oligonucleotide Conference, Neuromuscular Session Summary
Sharon Hesterlee (PPMD) (h/t @sport234a)
Prosensa $RNA IPO Prospectus SEC form S-1
Eteplirsen for the Treatment of Duchenne Muscular Dystrophy
Mendell et al. Annals of Neurology, 2013
Phase 2b clinical trial publication for SRPT.
Gene Therapy With a Difference
Andrew Pollack (New York Times) (h/t @given2tweet)
Recent Sarepta Therapeutics SRPT articles
SeekingAlpha (various authors)
Clarity of Subpart H
Christine McSherry (Jett Foundation - @fundduchenne)
GSK and Prosensa announce primary endpoint not met in Phase III study of drisapersen
Sharon Hesterlee (PPMD)
The Arguing Over PTC124 and Duchenne Muscular Dystrophy
Derek Lowe (In the Pipeline - @dereklowe)
Pharmacokinetics and Safety of Single Doses of Drisapersen in Non-Ambulant Subjects with Duchenne Muscular Dystrophy: Results of a Double-Blind Randomized Clinical Trial
Flanigan et al, Neuromuscular Disorders, 2013
Jack and Nolan Willis - 2013 MDA Telethon Profile
The "non-ambulant" twins on the eteplirsen clinical trial (h/t @jennmcnary)
Sarepta To Seek Early U.S. Approval For Muscular Dystrophy Drug (Updated)
Adam Feuerstein (The Street)
Sarepta Falls on Investor Skepticism of Approval Plans
Samuel Adams (Bloomberg)
Sarepta to Seek FDA Approval For Duchenne Muscular Dystrophy Drug
Ben Fidler (Xconomy)
Innovative Muscular Dystrophy Drug Edges Closer To Market
Matthew Herper (Forbes)
Which Drug Will Be the First Approved for Duchenne Muscular Dystrophy?
Rare Disease Report
For the Long-Term Investor, Sarepta’s Immediate Events Matter Little
Jake King (PropThink)
Profiles in Long-Termism: Sarepta Therapeutics CEO Chris Garabedian
Luke Timmerman (Xconomy)
Benefit/Risk Survey Results to Be Submitted to FDA with a Goal of Accelerating Review of Duchenne Therapies
PPMD