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The Pirfenidone protocol is on its way!

This week the 30th person in the phase III Pirfenidone trial to treat the pulmonary fibrosis of Hermansky-Pudlak Syndrome was enrolled. Yahoo!!!!! This is a big deal! We’ve been working on it for a very long time.

What does this mean? It means that the statistical clock is ticking. We still need 40 patients to complete the trial, however, we have reached the threshold at which we have enough patients to start the clock for the interim statistical analysis. At roughly 18 months, or half way through the trial, the statisticians will review the data thus far. If the patients on the drug are doing significantly better than the patients who are on the placebo, then for the next 18 months of the trial everyone will be put on the drug. That’s great news for those of us that are in the trial. It’s also great news for everyone else because it means we’re that much closer to getting the drug approved (if it works) and moving on to the next step to find the cure.

At the American Thoracic Society the doctors in Japan presented their data from their Pirfenidone trial. It was the one session I really, really wanted to see. I only got to see the last part of their presentation. Unfortunately, Karen had a bit of trouble and needed my help, so I missed their presentation. Donna assures me that she saw it and it’s all okay. “The stuff is working,” she said. But, I still would have liked to have heard the data for myself as well as any questions the doctors might have asked that I might have never thought of. Oh well – hopefully that data will be published very soon and we’ll all be able to read it.

In the meantime, continuing with the 100 people search is as critical as ever. We need to get people properly diagnosed, and we need to find the final ten patients for this trial.

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