...I feel like there is so much else involved in this, than just this one boy. . .
Will his part of the trial be different? How does this affect all boys involved in the trial?
Its Mark here from the UK - new to the forum. I think as long as this is treated as a seperate study - rather than being incorporated into any double blind random testing - they should be able to isolate any anomalies in the data arising from the inclusion of this additional patient.
Whether or not this is compassionate, do we know if it can be treated as a trial of its own? Would the FDA agree to this, given the circumstances. I don't see that the family would be concerned with that - in fact, they might they are not getting a placebo.