of the DMD disease ...pure validation
Sarepta Therapeutics Announces Eteplirsen Demonstrates Stability on Pulmonary Function Tests through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy
“My name is Jenn McNary, I have two sons with Duchenne. Austin is 15 and Max is 12. We are not so much in a race against the science as we are in a race against the clock. We absolutely will find a treatment for Duchenne- but will we find it in time for the families sitting in this room. In my home , I am faced with a stark reality on a daily basis, I have one son who is on Eteplirsen, and one who is not. While Max gains independence, Austin is becoming totally dependent- at this moment he is losing his ability to feed himself. We have a unique opportunity. This generation of children could be the last generation of children to die from or the first generation to survive Duchenne. We are that close. But it will only happen if the FDA does the right thing, listens to the researchers, examines the science and operates under the provisions of FDASIA. We are asking Congress to exercise its oversight of the FDA and to make sure the FDA is using tools like the Accelerated Approval pathway the way Congress intended, for treatments like Eteplirsen. The time is now. Please take this information back to your Members of Congress and your Senators, and be on the right side of history. If you act, you can be the hero of this story, thank you”.
we know what that will be ,the shorts will get caught up in the truth and suffer for what they are doing.
it is just a matter of time before justice avails for this company and it's science.
Remember this as well.
This drug could be approved under the animal rule.....easy...
solid long term growth with drugs that have no comparison to others.............bullish ,bullish long term buy
Sentiment: Strong Buy
the FDA for holding back this drug and comparing it to another drug among other harsh words.I understand those parents frustrations somewhat.
something else to think about'' The FDA passed words to Sarepta leading them on to release information that caused these class action suits therefor dragging then into the middle and this further drives this early approval .If you add all the positive drug effects with safety and lawsuits ,we have a more motivated drive here....
They have the power to get the FDA leaders replaced and or directed in the right path or else...Makes no sense to have this drug held back because the health and life saving effects along with the safety profile out weigh all other aspects by 100 to 1......................................We are talking about slow suffering and death without this drug in use.You could compare this to a slow cancer..........
anyone with good common science sense can see this drug is a ace............
Primary Outcome Measures:
The primary efficacy endpoint will be the change from baseline in the percent of dystrophin positive fibers in muscle biopsy tissue as measured by immunohistochemistry (IHC). [ Time Frame: 20 Weeks ] [ Designated as safety issue: Yes ]
The primary efficacy endpoint will be based on the pre-treatment and post-treament percent of dystrophin positive fibers as measured in the muscle biopsy tissue on(IHC).
Secondary Outcome Measures:
The secondary efficacy endpoints will be the change from baseline in: CD3, CD4, and CD8 lymphocyte counts in muscle biopsy tissue; 6-Minute Walk Test (6-MWT) distance. [ Time Frame: 20,80 Weeks ] [ Designated as safety issue: Yes ]
A key secondary efficacy endpoint will be based on the pre-treatment and post-treatment CD3, CD4, and CD8 lymphocyte counts as measured in the muscle biopsy tissue as well as a 6-Minute Walk Test(6-MWT)distance.
Study Start Date: February 2012
Estimated Study Completion Date: July 2015
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AVI-4658 (Eteplirsen)
Multiple-Dose Extension Study
Drug: AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion over a period of at least 60 minutes. Their are two treatment groups, 30 mg/kg and 50 mg/kg.
February 6, 2014
SRPT Advocates set to meet with Congress on Sarepta DMD drug
An advocacy group, along with a panel of Duchenne’s muscular dystrophy researchers, is set to meet with U.S. lawmakers tomorrow to urge accelerated approval for Sarepta’s (SRPT) eteplirsen. WHAT’S NOTABLE: According to a report in the Boston Business Journal, The Jett Foundation, an advocacy group organized by families affected by Duchenne’s muscular dystrophy, is set to meet with U.S. lawmakers tomorrow to urge accelerated FDA approval for Sarepta’s drug. The Foundation’s government affairs team will also be joined by a panel of Duchenne’s researchers. The Foundation said a similar meeting last year spurred 30 congressman to write the FDA urging fast action on eteplirsen. Sarepta said it is not involved in Friday’s meeting. ANALYST COMMENTARY: In a note to investors earlier today, analysts at RW Baird raised their price target on shares of Sarepta to $35 from $22 following updated data from the company’s Phase IIb trial reported yesterday. The firm said the update on pulmonary data provides incremental evidence of efficacy following the 120-week, 6-minute walk test data reported last month, which showed a general stabilization of walking ability in eteplirsen-treated patients. Baird maintains an Outperform rating on shares of Sarepta.
that is very possible soon.