If you look here there are testing dystrophin positive fibers at week 24 ''tell all''
you can't expect real walking benefits until after 24 weeks of dosing so walk preserving after 36 week would be the natural history change.''change of natural history of decline''
but the real deal is creating dystrophin that was not there after 24 weeks and the others have very little''
Primary Outcome Measures:
Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
The percentage of dystrophin-positive fibers [ Time Frame: Baseline, Weeks 24/48/TBD ] [ Designated as safety issue: No ]
Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ] [ Designated as safety issue: No ]
Estimated Enrollment: 160
Study Start Date: September 2014
It made no sense what ever ''sarepta is in a 160 boy trial and data has been given to the FDA constantly .
From the new 160 boy trial..will settle all and stock should be 72.00 and once approved over 100.00
Do you think it is possible that the FDA may be thinking of approving Eteplirsen under FDASIA before it even gets there? it is a good drug for children for unmet medical needs and a rare disease.
it always does......Sarepta's dmd drug is very far more effective than biomarin's . I would not even compare the two.
worth much much more.............................................................................
The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has tentatively scheduled review dates of November 23 and November 24 for drisapersen and eteplirsen, respectively. The former drug has already received an approval-decision date of December 27.
Sarepta recently said it was looking to buy a drug to treat DMD. Observers believe that the reasoning behind this would be to potentially mix the new medication with eteplirsen for a cocktail of treatments.
Why are you claiming Drisapersen is a effective treatment for DMD ? Almost every boy in your past trial was very young and by natural history would not be expected to have a decline.Are you only expecting a approval for boys under the age of 8 years old? If so where are the Dystrophin production proof long term? This is the very underlying reasons for dmd ''the lack of dystprophin''.
hopefully it will extent a longer life with improved quality.
An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients with Advanced Stage Duchenne Muscular Dystrophy (Protocol Number 4658-204)
Principal Investigator: Kathryn Wagner
This research study is being done to find out if eteplirsen can help people with advanced Duchenne Muscular Dystrophy (DMD). A predecessor trial has been IRB approved (IRB00040305). DMD is caused by a mutation in the gene that makes dystrophin which is important for protecting muscles from stress and damage during activity.
This research study will test whether eteplirsen works to improve muscle function in people with DMD who have deletions that may be corrected by skipping exon 51, and to find out if eteplirsen is safe to take without causing too many side effects.
Eteplirsen is designed to “skip” the exon 51 of the dystrophin gene. For people who have deletions in certain parts of the dystrophin gene, skipping exon 51 might potentially allow the body to produce a shortened, but still working, form of the dystrophin protein.
This research study will test whether eteplirsen works to improve muscle function in people with advanced DMD who have deletions that may be corrected by skipping exon 51.
getting kinda transparent now......................................................
FDASIA is a commitment between the FDA, industry and patients; thus, success its success is not only an achievement of the FDA, but a combined effort among all three. On the third anniversary of the passing of this law, it is clear that progress has been made to achieve this success and will continue to be made.
CAN'T BELIEVE THAT IS TRUE.. THE WHOLE CAUSE OF DMD IS ''LACK OF DYSTROPHIN''
THE WHOLE CAUSE'' IF YOU REPLACE ENOUGH IT WILL EASE THE PROGRESSION OF THE DISEASE''
Sarepta can trade Biomarin that voucher for their patents and ''give up'' that would be a easy way out for biomarin
i can't see how you can pimp a drug that will cause decline for monies''and you know it and live with it''
they fell off a turnip truck and hit their head or they know nothing about investing in this field .
The only real factors here are dystrophin production and real safety because of long term dosing..facts....