" slowing of progession of disease in mice is the basis for expecting small but significant chance of brain improvement."
But this doesn't address Charlies case which is the (supposed) additional brain damage caused by his epilepsy (epileptic encephalopathy as referred to in previous judgement). The slowing of progression in mice, as I understand it, refers to different variants, and note slowing of progression.
Okay, so my non-medical understanding is that what the NY witness is primarily talking about is at the cellular level. They feel that they have evidence that the muscular effects can be improved, hence coming off ventilation a possibility. The 10% figure referred to that. That since they also have evidence as regards passing the blood-brain barrier, it could also improve the function of brain cells. But, functioning brain cells are not a functioning brain. That doesn't address the additional factor of the 'catastrophic' brain damage caused by epilepsy. Would appreciate if someone biological could explain the structural/functional bit for brains.
Latest:
"would you be prepared to come to London and examine Charlie if I adjourned the case? Answer: Yes."
So, NY witness admits they cannot assess the amount of possible brain improvement without coming to assess the amount of structural brain damage. Or do they mean that they personally are not qualified to assess that? I assume the former............
What I'm thinking here is that even if there were cellular improvement in terms of muscles and breathing but minimal or no improvement as regards brain function, then you might have Charlie being 'beyond experience', but able to breath unaided. So then he would either continue like that, or decline slowly (let's not forget the general decline that GOSH referred to in the position statement as I recall....), or you would remove the therapy, wait for him to decline to where he is now, and not ventilate and allow him to die.
If a child had the muscle issue but still retained some brain function, then I would agree that the therapy to extend the quality of life they already had would be worthy. As was the case for GOSH before the catastrophic brain damage from epilepsy. But when we have the additional factor of the supposedly disputed 'catastrophic' brain damage, we in some cases have what I feel is a worse scenario, and that may have a higher likelihood than the best case (improved muscle and improved brain function).