|
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~
The Stop ALD Foundation Annual Update
December 2009
You helped us achieve a BREAKTHROUGH
in 2009! What's next for 2010?
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~
|
Greetings!
What great news we were able to deliver in
2009! While working hard in many areas, we are, by far, most proud of our groundbreaking international success in the field of gene therapy. This is the first time ever that a fatal brain disease has been successfully treated by gene therapy, and it was ALD! Our
published gene therapy success made news around the world in the scientific and
mainstream press. In case you have
not seen the details, we'll give a recap below. With your generosity and our hard
work, we pulled off what most foundations only dream of -- delivering a safe and
effective therapy for a disease. There is still critically important work ahead
for 2010, and we'll tell you about our goals and how we would greatly
appreciate your continued support.
Gene therapy breakthrough!
 On November 5, 2009, the
two French researchers with whom we have worked closely had their
pioneering findings published in Science. In this groundbreaking paper, they describe two boys
with ALD who received gene therapy treatment and consequently had their brain disease progression halted, and some
damage was partially reversed.
This biomedical advance is significant beyond the realm of
ALD. We would like to extend our
heartfelt thanks and gratitude to you, our supporters and generous
donors. By delivering this new treatment, you have helped to make a difference in the lives of
children and adults suffering from ALD and AMN globally. In addition, the approaches utilized are applicable in the treatment of many other diseases, such as metachromatic leukodystrophy (MLD), Wiskott-Aldrich Syndrome (WAS), Hurler Syndrome, Krabbe disease,
and more. You should be VERY proud.
|
