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HemaQuest Announces Initiation of Phase I Clinical Trial of HQK-1001

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Manal:
Dore, actually i don't know but this medication is supposed to help in inducing the fetal HB instead of the adult HB ( in thal you are not able to produce adult HB but fetal HB can compansate) so if the enzyme you miss (pyruvate kinase) is also important in maintining the fetal HB too, so i guess it won't help.

I wonder why this enzyme is not artificially produced and is taken as shots :huh :huh :huh, do you have any idea??

manal

Dori:
No, I have no idea. On 4th October 2008 died mr. Beuthler. He was our KING of this disease. Sadly, we have (could) contact him. So I hope there are others left who will continue his work. There has been one Dutch chemist (smth like that) who did also reach on pk def. He found 60 people with pk def. in my country, and I only 6 so far. I often think about that too. Pk def. is pretty rare. Only one person; 5-yr old child from Vietnam has had a bmt. It was a succesfull one; they said. Btw the other members of his family have....thalassemia!

Dore
p.s. lot's of cats and sometimes dogs has pk def. as well.

Sharmin:
Dore,

I am curious about whether Pk def. affects fetal hg.  I ask this because fetal hg is required before birth and during the first few months after birth - therefore perhaps the Pk def. does not affect fetal hg and in that you could benefit from something like hemaquest.

At what age did you become anemic and begin to require transfusions?  I could be wrong - but it would be nice if you could also benefit if something like this become a reality,

Sharmin

Dori:
It does not affect fetal hemoglobine. To come back at the other question: giving injection of pyruvate does not help. The pyruvate must be get inside the blood cell, otherwise you can not fix the problem. That's what my father told me. He said we gonna try a less iron diet btw. And he had an other plan. We'll see what happend.

I had a transfusion on the same day I was born (in 24hours). I was born 1 month too early though. It was an exchange blood transfusion. That stupid human who did it DESTROYED my own blood. That's why it took sveral years to find out what kind of anemia I had.

Dore

Manal:

--- Quote ---It does not affect fetal hemoglobine
--- End quote ---

Dore, since it does not affect the fetal Hb why don't you discuss with your doctor taking fetal HB inducers like hydroxurea??

manal

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