[Jack]

Hi all,

Parent of a 12 year old FA-er, who currently takes Lioresal, melatonin, and…that’s all. I’m not a biologist, but I routinely read and write patents in my field, so I thought I’d give this one a try.

From what I can read in the patent, the dose span is pretty large as it is probably targeting trials, and shouldn’t be taken as a recommendation. At least not from me!
The Italian team claims the following test doses for adults (over 18)
– between 100 and 300mg, twice daily
– 200mg twice daily
– between 50 and 150mg, twice daily
– 100mg twice daily

and for children (6 to 18) (see table 1 on page 6)
– 16 to 20kg: 100mg twice daily
– 20 to 25kg: 125mg twice daily
– 25 to 30kg: 150mg twice daily
– over 30kg: 200mg twice daily

The most striking is that the dose you guys take (2x100mg, as far as I could read, ie. the recommended dosage for HIV+ patients) is lower, by a factor of 2, than most doses Testi et al. intend to test. It is highly probable that they derived the target daily doses from older HIV studies that examined how etravirine gets absorbed by patients, and targeted the in-vitro concentrations they found most effective on patient-derived cells.

I’ll be following this thread as it seems the most promising medication so far. My gut feeling is that the molecule is not a cure-all medication, and will rather be part of a combination therapy at some point in time. The existence of an “opportunity window” in FA seems more and more likely, so I hope children will be involved soon in trials (but I know it’s unlikely they will at first).