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Pat Girondi Interview and Update

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InGreece:
I agree with everything being mentioned above. Affordability is a major issue but I'm optimistic that if a comprehensive therapy is developed it will be bought and distributed as most countries will save money even in the medium term.

One more thing about the interview:
Mr. Girondi is great and his passion immediately comes across. It's this can-do mentality that makes things happen.

I just have one question, which is -I''ll be the first to admit- kind of heavy : I heard him discussing mortality rates. My impression has been that given proper care (regular chelation, proper monitoring of iron deposits) thal children today (and even an 18 year-old like his son) can live lives that have a normal life span. In Greece Thals get married, have children. Certainly this was something that we were told from our doctors. And not just "some will make it" but rather that given the state-of-the-art currently the expectation is that there should be no noticeable difference in life expectancy.

I'm only bringing this up because our (my wife's and my own) hearts kind of sank when we heard the discussion about life expectancy in the thirties. I was under the impression that this was 20 years ago.
Could it be that he was stressing the problems because audiences wouldn't get the importance of managing a genetic problem if they heard people with it can live full lives? I would understand.

I hope so. If not, then we should be doing a reassesment of how we are looking at the long term strategy of managing our daugher's care.

Sorry to bring this up.
And if anything it shows how imprtant people like Pat are, who give us hope via their work.
Our prayers are with him.

Andy Battaglia:
I think the average life expectancy figures we hear are worldwide and include people who get little to no care. With proper treatment, thalassemics should live fairly long lives now. Enough so, that several years ago in the US, thal was reclassified as a chronic rather than a fatal disorder.

Sharmin:
Hi InGreece,

During our visit to Oakland Dr. Vichinsky mentioned that he has 50 year old patients with thalassemia major who are doing very well.  Remember also that these patients did not have the level of care that our children have as desferal was not available for some of these patients until much later in life.  The outlook for our children is good as long as we stick to a good treatment regimen.  Chelation is the key to survival and to the avoidance of complications. 

In light of some of the new research that is taking place the future is promising for our kids.  If gene therapy is successful - which I am hopeful that it will be - then our children may be cured.  For patients who have antibodies making their own blood and avoidance of exposure to foreign blood should alleviate the hemolytic anemia as well - let's all hope that gene therapy will be successful and available soon. 

Sharmin

Canadian_Family:

--- Quote from: Andy on January 07, 2009, 09:05:31 PM ---I think the average life expectancy figures we hear are worldwide and include people who get little to no care. With proper treatment, thalassemics should live fairly long lives now. Enough so, that several years ago in the US, thal was reclassified as a chronic rather than a fatal disorder.

--- End quote ---

Thank you Andy.

InGreece:
Thank you Andy and Sharmin.

If anything this highlights the importance of making proper chelation available to people in countries where the health system is less generous.

This is indeed a worthy cause.

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