6 children with rare genetic disorders show no sign of disease after gene therapy.
No doubt patient advocate groups will get these treatment to patients even if BigPharma continues to ingore them. Interestingly, the company concerned got fed up with begging for scraps and started raising money via telethons to get these treatments into patients. Looks like their gamble is starting to pay off.
The dam wall is beginning to crumble where gene therapy is concerned. They just need a non-orphan disease cure to make people wake up. TT-034 could be the one.
