is a very postive life extending outcome for this agressive disease.If you add any years of living life you have done a wonderful outcome.
We know the Sarepta dmd drug works better than any other ''even if long term it is only 25 percent effective ''we do know with curent short term it is about 55 percent effective''either way this drug will be a blockbuster drug.Once we get the sales and monies come to bare we can then start many other trials that will avail the same.
It is my opinion that exon skipping drugs should be dosed daily for having the best effects.
Isis Pharmaceuticals, Inc. (ISIS)-NasdaqGS Watchlist
68.59 3.15(4.81%) Jan 7, 4:00PM EST
|Pre-Market : 71.25 2.66 (3.88%) 8:43AM EST
They don't have enough people for a class action suit for one and two these same law firms are doing the same to 275 other companies.they are trying class action because they could not ever win against a delaware corp.
•Male 7-16 years old
•Diagnosed with DMD, genotypically confirmed
•Stable dose of corticosteroids for at least 6 months
•Have intact right and left alternative upper muscle groups
•Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters)
•Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%
•Previous treatment with drisapersen within the last 6 months
•Participation in any other DMD interventional clinical study within 12 weeks
•Major surgery within 3 months
•Presence of other clinically significant illness
•Major change in the physical therapy regime within 3 months
This is an open-label, multi-center, 48-week study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group).
Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg Eteplirsen. Patients in the untreated group will not receive treatment.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.
Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.
The sponsor is working to initiate approximately 39 sites across the United States. Sites will vary in the following functions:
1.Local Site (N=39) - Enrolls patients and is the primary contact point for their patients. Sites will perform all protocol activities (including dosing and laboratory assessments), except for functional assessments and biopsies.
2.Hub Site (N=14) - Performs functional (physical) assessments at specified times per protocol.
3.Surgical Site (N=2) - Performs muscle biopsies for the Treated group.
the year of small caps/
Look at Sarepta's science and see that it is 100 times better '' isis has second generation antisense and sarepta has third generation and beyond .A smart third grader could see this.
1 Recruiting Confirmatory Study of Eteplirsen in DMD Patients Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: eteplirsen injection
2 Recruiting Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: eteplirsen
3 Recruiting Phase I/II Study of SRP-4053 in DMD Patients Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Placebo; Drug: SRP-4053
it would be stupid to paint any other picture.
It is also my opinion after 6 months of dosing the drug should be given every 3 days not every 7 days or every 3 days from the start.
no matter what.....also no other drug as good or better in the pipeline near term ,this makes Sarepta's drug a blockbuster.