Forbes: In A Dramatic Result, An Experimental Drug May Help Boys With Muscular Dystrophy

[PiLfan]

http://www.forbes.com/sites/matthewh...lar-dystrophy/

A new experimental drug appears to improve the muscle function of boys with muscular dystrophy, increasing the distance the boys can walk in six minutes compared to when they began taking it a year before.

Those results are “incredibly promising,” says Lee Sweeney, chairman of physiology at the University of Pennsylvania’s Perelman School of Medicine, who studies muscular dystrophy and was not involved in the study. “They don’t ever improve once they start going down. The fact you can improve them is not something that happens normally. That’s not random.”

The most common form of the disease, Duchenne muscular dystrophy, affects only 13,000 American boys; girls almost never get it because is caused by a defect of a gene on the X chromosome, of which girls have a second copy. The new medicine, eteplirsen, is targeted at the 13% of those boys whose muscular dystrophy is caused by a certain type of mutation. Without treatment, patients’ muscles continually weaken, leaving them wheelchair-bound; they frequently die by their 30s.

Eteplirsen, if successful, will be a huge victory for muscular dystrophy patients and researchers, who have never had a treatment that might make any of these kids get any better.

This is awesome news. DMD is a horrible, horrible disease and it looks like we are that much closer to a cure for it.

[China]

This is clearly a rare disease and this treatment is for an even smaller subpopulation as it is only treating 13% of 13,000 or about 1700 boys. Hopefully it will help them develop treatment(s) for the other MD patients. Regardless, this is good news and I'm sure the parents of these kids are thrilled that there may be some hope on the horizon.

[PiLfan]

The good news is that while this particular treatment is for a 13% subset of the population, the drug's technology can be used to deliver to the other subset types as well (known as 'exons'). The exon this treatment is designed for is one of the larger ones...there are many different types, but the techonlogy should work for most if not all, since the basis of the disease is the same (lack of dystrophine) due to a gene abnormality, and the treatment platform is designed to 'skip over' the malfunctioning gene and allow for the necessary muscular production.

Still a ways to go before this can be labeled as an outright cure for all of DMD, but it's by far the most promising thing to come along to date. A win for Jerry's Kids, indeed.

[No_Pressure]

As I think "hey this might be a good stock buy if its publicly traded" I looked it up. It's gone from 14.99 to 37 so far today. 150% increase. FFS why can't I get up earlier in the morning?

[China]

As I think "hey this might be a good stock buy if its publicly traded" I looked it up. It's gone from 14.99 to 37 so far today. 150% increase. FFS why can't I get up earlier in the morning?

Interesting, because if this drug is approved, I doubt they make much if any profit on it. For that small of a patient population, even though they can get some Orphan Drug protections/incentives, the cost of development and production will likely outweigh whatever they get for it once approved due the small number of people that will buy it.

[No_Pressure]

Interesting, because if this drug is approved, I doubt they make much if any profit on it. For that small of a patient population, even though they can get some Orphan Drug protections/incentives, the cost of development and production will likely outweigh whatever they get for it once approved due the small number of people that will buy it.

Yep. They're hovering around 40 now http://finance.yahoo.com/q?s=srpt&ql=1

I'm sure it'll go back down, perhaps even fall quickly tomorrow or later in the day. Sometimes these companies just surge for a day or so on the news, and drug companies are a popular surge buy for whatever reason.

The one I'm waiting on which I bought is VICL (Vical Inc.) I got in at about 3.30 or so, it's at 4.30 now, but they're making a miracle cancer drug which is finishing up some trials in November I believe and if the news is highly encouraging I expect this sucker to take off. Talk about a large patient population!

[China]

If Allovectin is approved, it would be the first gene therapy approval in the US.

[pjfootballer]

Jeez, they may have found a good thing to cure something and just like the pharma companies, the only thing people can think about is money. This is what's wrong with our country and healthcare.