Well, here's a story that I wasn't expecting to have to write. If you've followed the Pompe story through this blog (and if not, why not?), then you'll know that there have been occasional patches of...murkiness. Times when things have happened that the reader may have had to stretch to see in a good, or even neutral, light.
Nearly all of those have been concerned with commercial interests and, well, I think you kind of expect it from that quarter, where the bottom line reigns supreme. At least, I thought, the same could not be said of the world of academic science. A world where truth and objectivity were tirelessly in the service of the scientific ideal. You know, the good guys.
Turns out, I was wrong. There are significant errors - errors which are difficult to explain - in the original publication of the ERT trial results from Duke University, one of the most important publications in the development of ERT.
The publication in question is:
The full text is available online.
Over the next few posts, I will spell out exactly what the issue is, why it matters and what I think should be done.
Nearly all of those have been concerned with commercial interests and, well, I think you kind of expect it from that quarter, where the bottom line reigns supreme. At least, I thought, the same could not be said of the world of academic science. A world where truth and objectivity were tirelessly in the service of the scientific ideal. You know, the good guys.
Turns out, I was wrong. There are significant errors - errors which are difficult to explain - in the original publication of the ERT trial results from Duke University, one of the most important publications in the development of ERT.
The publication in question is:
Recombinant human acid a-glucosidase enzymeAmalfitano et al Genetics in Medicine, 2001:3(2):132–138.
therapy for infantile glycogen storage disease
type II: Results of a phase I/II clinical trial
The full text is available online.
Over the next few posts, I will spell out exactly what the issue is, why it matters and what I think should be done.
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