Charity Navigator, which is the largest independent evaluator of charities, gives MDA two out of four stars based on Financial, Accountability, and Transparency Performance Metrics.
mitrochondrial DNA or some other genetic mediated disease
The kids and their parents shouldn't have been there. SRPT turned this into a public relations circus and the FDA allowed it. The only thing that matters is the data. This story reminds me of Dendreon whose drug didn't work, but was approved due to outside pressure. The ultimate power behind Dendreon's lobbying efforts, like SRPT's were those who were going to make money.
The business of selling false hope is a great one on wall street
the sec and us attorney should take a close look at sarepta....preying on the most vulnerable group of pediatric patients to make millions,,,,a 12 person study?
no cars in parking lot on Saturday....looked like a ghost town.....Vicor just down the street booimg - parking lot was full on saturday lots of O.T .
your statement that you wouldn;t bet against a stock that helped people is the crux of the argument, the only people being helped here appear to be the insiders and investment banks that are lining their pockets by selling false hope.
no science behind its trial
sold me tires that wore out in 12,000 miles than the manager was never there when I wanted replacement.
now I won't spend any money at sears even if everything 50% off....that's my contribution to sears bankruptcy
many small biotech companies floating stock and taking in billions of investor money, but the companies never deliver an approved drug.. Selling false hope of a miracle cure is just like junior mining companies selling the dream of a big gold strike.
There seems to be a trend where drug companies develop new drugs simply to have products to sell. After developing these drugs many are advertised on TV like dish soap or some other ordinary consumer product. The ads are psychologically manipulative - with happy scenes of people cuddling puppies or dancing at a family cookout while the ad's narrator drones on about endless side effects that can be worse than the disease.
Equally symbolic of a ethics system that's broken are the developmental stage companies that see people's illness as a gold mine and try to strike it rich by floating stock and selling the dream of a cure. These stories of hope drag on for years with company insiders and wall street bankers making fortunes before sobriety hits. Yes people should be compensated for their inovation and developmental costs, but the current compensation structure is rife with stock swindles based on selling false hope. Morally and ethically this is not right.
4045-301: Phase III Study estimated enrollment 99....is not yet open for enrollment........no other phase III.........
If they have more data than the 12 person study I'd be interested in knowing. Rather than spouting off at the mouth put up the reference or shut up.
I just read the BostonBiz article on SRPT. The problem with the article is it didn't mention SRPT's clinical data is based on very few participants. In the 201 trial only 6 individuals got the drug and and 12 received it in the 202 trial. The drugs efficacy is questionable. I quote from the FDA briefing document: "Figure 2 shows the change over time in NSAA scores for all 12 eteplirsen-treated patients in Study 201/202. All show progressive declines in NSAA scores, with six patients moving to NSAA scores that have been reported to be associated with being within one year of loss of 1 Mazzone E, Martinelli D, Berardinelli A, et al. North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy. Neuromuscul Disord 2010; 20:712–16. ambulation, and an additional four patients moving to scores associated with being within 2 years from loss of ambulation."
As an investor I see too much risk and uncertainty in this stock. If I had to wager I'd bet on non-approval. The SRPT story reminds me of Dendreon back in 2007 when people campaigned for the approval of its drug. From the little I know, SRPT looks suspect.