PER - Megathread, page-275

The families dont have an easy choice, do they?

This has always been a chicken or the egg type treatment. Treat the gene deficiency to stop the inflammation and allow the muscle to repair? Or treat the inflammation and give the gene therapy a cleaner environment to work on?

Lots of money has been spent on the gene therapy route. As inflammation has been thought to be the byproduct of the gene dysfunction. So root cause treatment was considered to be fix the gene! Trial data, well PER trial data, would tend to suggest the reverse may be true (well, it will if PER can replicate results from its last trial). There is thought, but not yet trialed, that the answer may be in the middle.

The Tox study, the biomarkers, the interaction with exon skipping drug, etc. work that has been done, all
all could drive ATL1102 along many different pathways.

And then there is all the other CD49 inflammatory diseases.