What Is Hemophilia

Sangamo Shows Early Promise Against HIV, Hemophilia at Gene Therapy Revival ...

Word on the street in Seattle last week was that Big Pharma was sniffing around quite a bit at the American Society of Gene & Cell Therapy annual meeting being held downtown. There was a lot more optimism at this confab than in years past, and Richmond, CA-based Sangamo Biosciences was one of the companies capturing some of the buzz.

Sangamo (NASDAQ: SGMO ) had 20 different presentations of data, mostly of the early/preliminary kind, at this meeting. Scientists were there to hear more about Sangamo’s proprietary “zinc-finger protein technology,” which is designed to edit specific DNA sequences inside a cell, to, say, turn off a disease activity. Many investors are waiting to see if Sangamo can deliver the goods on this concept later this year when results are due from a trial of an experimental drug of this variety in 170 patients with diabetic neuropathy—a big potential application of the technology. But most of Sangamo’s presentations at the gene therapy meeting in Seattle focused on using its technology in a number of other diseases, like HIV, hemophilia, and retinal neurodegeneration, to name a few.

Most gene therapy approaches, for those who don’t follow the field every day, involve modifying viruses to carry copies of genes into cells where they can replace missing or faulty genes at the root cause of certain diseases. Gene therapy was hyped in the early 1990s as a potential cure-all for all kinds of ailments that have evaded traditional drug-based approaches. Safety concerns have popped up more than once, and there still aren’t any FDA approved gene therapies, although there have been some encouraging results lately that have sparked a bit of a comeback.

“I’ve been coming to this meeting since 1990,” Sangamo CEO Edward Lanphier said during a break at a downtown hotel on Friday. “It goes up and down in terms of what’s hot and what’s not.”

Here’s a couple key programs that Sangamo highlighted at the conference, and which I discussed with Lanphier.

—Sangamo scientist Michael Holmes presented data from a study in mice which showed encouraging results in a specific type of hemophilia. In this case, the disease comes from a deficiency of a protein known as Factor IX. Scientists gave a single injection of one of Sangamo’s zinc-finger compounds and found that mice who got the injection were able to produce Factor IX on their own in high enough levels to achieve normal blood clotting, Lanphier says.

Hemophilia: What are the symptoms, diagnosis, genetic patterns of ...

Are easy bruising, prolonged bleeding and in some cases can lead to death. Most hemophiliacs fall under the heading of Factor 8 ( hemophilia A) or Factor 9 (hemophilia b). The factor simply put is the clotting that they are lacking. Factor 8 is the most common.

It is important to remember that like many things there is not just one type of Hemophilia. Some hemophiliacs may only need for a major injury or surgery. While others may get muscles and joint bleeds and not know how the bleed started.

Treatment:

Mild hemophiliacs may only get treatment when need to before a surgery. While those with severe hemophilia may treat 3 times a week regardless if there is a bleed or not. This is done to improve the quality of life. Many hemophilics are often taught to treat at home. This means finding a vein and sticking yourself with a needle.

Factor 9 hemophiliacs are often given Benfix. While factor 8 hemophiliacs are now getting Whety more often as it just hit the market in Jan 2008. Both are VERY VERY expensive.

Well you need to be a little more specific on which kinda, Hemophilia A (Classic) and Hemophilia B (Christmas) are completely different. Hemophilia B is very severe, although this type of hemophilia is far less common, the symptoms associated with hemophilia B is often what people think about when they think hemophilia in general. Although Most consider hemophilia as a disorder where people cannot stop bleeding, this is a huge misconception. People with hemophilia, whether A or B, stop bleeding, it just takes them a little longer, in the case of Hemophilia B, a lot longer, this is why it is the more severe of the two. Hemophiliacs with Hemophilia A (the majority of hemophiliacs) are not much different then we are. They can cut themselves they same way we can, and they might bleed for an extra 10-15 minutes, but the blood will eventually clot, just like us. No touching or patting either hemophiliacs will not cause a bruise. They receive bruises just like we do, only the bruises they get might be slightly larger, and stick around for a few more days. The because difference between a hemophiliac and someone who does not have it, is the internal bleeding, and while yes, it is possible that a sever blow to the head might cause death, this is true for everyone, and I have had many hemophiliac patients come in after an injury to their head, we give them their factor, and they can simply walk out. The only complete cure at the moment is a liver transplant. However, that isn’t exactly easy to do, so most hemophiliacs just receive treatments of their missing factors, depending on the type of hemophilia. People with hemophilia B receive factor 3-5 times a week, while people with Hemophilia A can receive factor anywhere from 3 times a week, to twice a month, depending on their lifestyle and how careful they are. When you think of hemophilia, you’re generally thinking of Hemophilia B, even though it is far far less common. Hemophilia A is not very crippling of a disorder at all, and most people who suffer from it lead lives exactly like people without the disorder, even keeping it to themselves, the misconceptions are not true, they can get cuts just like us, and heal, they wont die from a bruise, and a lot of them lead pretty dangerous lives(one of my hemophiliac patients is a professional skateboarder.) and they can get tattoos and piercing and all the stuff. The most important thing to consider. No matter which of the two hemophilia’s we’re talking about, in both cases, the bleeding does eventually stop, regardless of whether or not treatment is given. In regards to Hemophilia B, it takes much much longer, and may get out of hand, but in regards to Hemophilia A, I would just add another 10 minutes, maybe not even that. , and whether or not there is a cure (is there one being worked on?). Thank you for your help!

What is hemophilia?, symposium

Hemophilia

Horseman's veterinary encyclopedia

Textbook of Hemophilia

34th Hemophilia Symposium, Hamburg 2003 : presentation: HIV infection and epidemiology; management of bleedings in hemophiliacs with inhibitors; orthopedic problems and therapy in hemophiliacs; therapy with protein c; pediatric hemostaseology; free lectures