drisa is toxic and is high dose restricted and that is the big reason it does not work ,that chemistry has many off target effects that could cause many problems.''second generation antisense'' Sarepta has third generation antisense'' can give in large does safely'' no off target effects'' unless pints at a time''haa
It seems they are hiding that data because their drug ''being low dosing '' may make some but maybe it is causing other problems attached to that data????
This is now Biomarin's dmd drug,,At these ages water should have worked.
3. GSK/Prosensa clinical trial in DMD boys with study drug Drisapersen GSK2402968 (PRO051).
A multicentre trial with this study drug recruited DMD boys in UK at the Great Ormond Street Hospital(GOSH), London and at the Royal Victoria Infirmary, Newcastle. DMD114117 was a Phase lla, double blind, exploratory, parallel clinical trial to assess the optimal dose of GSK2402968 for safety, tolerability and efficacy, in ambulant patients with DMD. The primary objective of this study was for efficacy of two different dosage regimens versus placebo and the secondary objectives of safety, tolerability, PK and long term efficacy. It recruited up to 54 ambulant DMD boys from 5-7 centres in Europe aged at least 5 years and correctable by this study drug induced exon 51 skipping. These patients were recruited into a 24-48 month extension study(DMD114349).
The study was suspended on 23.09.2013 when initial data showed no favourable benefit.
2/28/2013 7:59:44 AM
Duchenne muscular dystrophy patients treated with GlaxoSmithKline's (GSK_) experimental drug drisapersen have been hospitalized due to kidney toxicity and low platelet counts, according to a Glaxo scientist who spoke at a research meeting in Rome last Sunday. Glaxo has disclosed some safety data from early-stage drisapersen clinical trials previously, mainly related to moderate cases of proteinuria (excess protein in the urine) and decreased thrombocytes, which play a role in blood clotting. However, the significance of the adverse events attributed to drisapersen, also known as GSK-2402968, escalated with Sunday's disclosure of hospitalized patients. Dr. Rohit Batta, global medical leader in Glaxo's neuromuscular rare disease unit, said four Duchenne muscular dystrophy (DMD) patients treated with drisapersen required hospitalization due to thrombocytopenia and that "several" patients with "severe proteinuria" also required hospitalization. Batta's remarks were made during a presentation on Sunday, Feb. 24 at The XI International Conference on Duchenne Muscular Dystrophy. The conference was held in Rome and organized by Duchenne Parent Project Onlus, an Italian DMD advocacy group. Batta provided no other information about the hospitalized drisapersen patients during his presentation. A Glaxo spokesperson contacted Wednesday confirmed Batta's remarks and added the DMD patients hospitalized were all enrolled in the ongoing phase II and phase III clinical trials of drisapersen. The hospitalizations had occurred "over the last two years" and had been discussed at previous medical meetings.
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Center, 48-Week Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy
MedlinePlus related topics: Muscular Dystrophy
Genetic and Rare Diseases Information Center resources: Becker Muscular Dystrophy Duchenne Muscular Dystrophy Muscular Dystrophy
U.S. FDA Resources
Further study details as provided by Sarepta Therapeutics:
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
This trial is around 11 months old....or 44 weeks .. should have 24 and some 36 week data..The old 12 boy trial is very old and still going.Keep in mind it takes 24 weeks for this drug to build up and produce dystrophin in boys,this is how the drug works.fyi
I am wondering how Biomarin will talk around real facts?
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....
i can't see how you can pimp a drug that will cause decline for monies''and you know it and live with it''
Sarepta can trade Biomarin that voucher for their patents and ''give up'' that would be a easy way out for biomarin
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''
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.
getting kinda transparent now......................................................
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.