Curing AMN: A Tall Order

Back in the old days, I was pretty sure that a cure for AMN was just around the corner.

I learned that there had been a transgenic X-ALD mouse model for many years, and I kept hearing about the grand gene therapy trials that were right around the corner. Still, things were not happening as I would have liked. At the time (mid-to-late 90s), efforts were limited to re-purposing old medications; not exciting. Still, I readily volunteered for the few trials that I could find. The ones that I remember tried lovastatin and a couple different ways to use 4-Phenylbutyrate.

I went into these studies with great optimism, and I guess, naïveté. I was certain that these tiny experiments of 12 people would work and turn my disease on its head. None of them did.

By this time, I had been in the pharmaceutical industry over 5 years, and thought that I knew pretty much everything. I guess the problem was, in my roles, I had it easy, the…

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