CAMBRIDGE, MA--(Marketwire - Oct 3, 2012) - Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its lead exon-skipping compound, eteplirsen, met the primary efficacy endpoint, increase in novel dystrophin, and achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT) over the placebo/delayed treatment cohort in a Phase IIb extension trial in Duchenne muscular dystrophy (DMD) patients.
Eteplirsen administered once weekly at either 30 mg/kg or 50 mg/kg for 48 weeks (n=8) resulted in a statistically significant increase (p≤0.001) in dystrophin-positive fibers to 47.0% of normal. The placebo/delayed treatment cohort, which had received 24 weeks of eteplirsen at either 30 mg/kg or 50 mg/kg following 24 weeks of placebo (n=4), also showed a statistically significant increase in dystrophin-positive fibers to 38.3% of normal (p≤0.009).
You have a lot of dystrophin production a certain way and the FDA was trying to measure it from a different way. you may want to try to understand completely before screaming 1 percent dystrophin because than is really not the only way the to measure at all.
They be crazy selling watch whats coming.......................
if they got any sense
It takes about 24 weeks to get a real good dystrophin production with Etepirsen and this is when the benefit starts so while taking this long ,we see positive fibers of 12 to 18 percent in muscle and therefor it would take about 5-7 years of dosing to get really going to the core. You could dose 2 times per week to create a faster outcome. For sure Eteplirsen works and you must start somewhere and improve as we go.
The Eteplirsen kid's Parents see the real life benefits plain and simple. ''also the real dmd experts see this as well''
Replace Janet Woodcock ...if she can't do any better than she has..' I am not impressed at all.
Watch your kids die slowly and see what you would do. Myself congress needs to be aware of the FDA's games.
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2016 by Sarepta Therapeutics
Information provided by (Responsible Party):
First received: April 10, 2015
Last updated: March 7, 2016
Last verified: March 2016
History of Changes
By Bruce Fisher -
April 8, 2016
Sarepta Therapeutics Inc (SRPT) : Ghost Tree Capital scooped up 90,000 additional shares in Sarepta Therapeutics Inc during the Q4 period, according to a recent disclosure to the SEC. The investment management firm now holds a total of 100,000 shares of Sarepta Therapeutics Inc which is valued at $1.8 Million.Sarepta Therapeutics Inc makes up approximately 1.05% of Ghost Tree Capital’s portfolio.
cover soon.......Sarepta's Eteplirsen really works , a real DMD expert would know and anyone that can understand the mechanism of the drug, know it works. Some people just don't know or understand ''even farkas'' This is a new era of medicines that targets what it is meant for and only that target'' no off target effects .
and if it goes up 100 pts in in year........wow
They like bribes , without that they drag it out to the end. ''what in it for me type of people'' cold hearts it seems'' we will see really soon