this drug from what I can tell is toxic... can't be dosed high enough to provide benefit and is clearly
beaten like punching bag by Eteplirsen.
Biomarin tried to go cheap..and you know you get what you pay for.
If I ever saw writing on the wall - this is it. This is a 20x40 BILLBOARD done in RED spray paint, surrounded by search lights and rimmed in ultra-neon.
Drisa is GARBAGE, we know it, the parents know it, the kids know it and most of all the kidneys know it.
"A recent example of the FDA’s indefensible inconsistencies is its October decision to demand more data yet again—without clear, science-based standards—for companies seeking approval for several new drugs that should be available now to patients with the fatal disease of Duchenne muscular dystrophy. What makes the muscular dystrophy pronouncements worse is that there are signs that they are driven by the FDA’s worst cultural tendencies: retaliation behind closed doors, “leveling the playing field” for competing companies by slowing down the company in the lead, and improperly relying on outside advisers with a strong financial interest in the outcome of its decisions. As the new Congress establishes its oversight priorities, this shameful mess should be high on its list.
The FDA should learn from its mistakes. It needs to be more proactive with guidance on the standards for approval of drugs for Ebola and other tropical diseases, more transparent about the safety and efficacy of investigational drugs for Ebola, and more understanding about why randomized trials for rare diseases are often not feasible. When it comes to children dying of muscular dystrophy, the FDA needs to show more of the urgency it displayed when approving investigational Ebola drugs for heroic public health workers. "
Nothing happens. In this instance - kids lose function and die.
What we know today is that drisapersen is toxic and quite frankly seems not to work.
The CEO of RNA stated "..the drug did not fail the trial - the trial failed the drug".
Now if you believe that tripe I want to sell you a bridge in Brooklyn.
I hope Biomarin sells dialysis machines.
Starfe.. rather than bombard this board with this boring and repetitive blather..complain to the company... no one here cares.
No one cares.
Some peeps LIKE Chris - nothing you say HERE will change that.
“This confirmatory study with eteplirsen in ambulatory patients is an important step in the process of confirming the promising clinical results we achieved in our previous studies 201/202,” said Edward Kaye, M.D., Sarepta’s Chief Medical Officer. “By evaluating a larger population of boys and examining both functional and biochemical endpoints, we expect to further support the evidence of eteplirsen’s potential benefit to patients.”