Discussion Forums > Working Towards a Cure
The Latest from Pat Girondi on the Progress of Gene Therapy
Andy Battaglia:
Baloch,
As Manal said, we can't know the answers to these questions until trials are done in people and the results are known. I will add that Dr Sadelain told us in Dubai that there is no definite age limit, but rather the health of the individual patient will be the determining factor. This is true will BMT also. Being in good health with no organ damage makes one a much better candidate.
Lena:
I also know that there is no age limit...although I am very pessimistic now that things will develop within my lifetime...as far as I am concerned, best thing is to stay healthy within thalassemia.
Lena.
Andy Battaglia:
I agree, Lena. We see how long each development takes to reach approval. Expect gene therapy to take even longer because of concerns about the long term effects. Until it has been tested long term in patients, I do not expect to see approval.
You are right. The tools are there to provide lives for those with thalassemia. Take advantage of what is here now and live a good life.
Lena:
As long as thals can benefit from tools available to ameliorate their daily lives, they must be happy and without stress, 'cause they must consider themselves to be part of the general population - inspite of thalassemia. Life limits are open now to thals as they are to any other people - but only if they comply with every known rule of how to treat thalassemia.
Lena
baal:
there is one think i mentiont...
since the 80`s they speak about the
gene therapie for thal`s.....
but nothing happend....
and now u says it start september....
i remer somethimk like Pat g. goes in spring 2010 to italy to start genetherapie....
now september.....
sorry but it sounds the same like the all the other storys forom the 80`s.....
i dont believe in GENETHERAPIE.....NO more
by the way ...im doing fine...panos
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