I started this group and discussion because my son Skyler, 5 has a confirmed premature stop codon on exon 11. He is eligible for the phase 2b clinical trial, and he seems to meet all of the other pre-screening requirements.
His mutation confirmation was a huge blessing since only 15% of DMD Boys have this mutation. But it is fast becoming a source of stress as well, as we try to decide on whether or not to enter him into this long and demanding clinical trial. It is 48 weeks long, with many invasive tests, including blood work every 3 weeks, and two muscle biopsies from bicep.
There is a chance that he will not even receive the drug over the course of the trial, as 1/3 of the 165 boys will get only a placebo. But he will still have to endure the full rigors of all the testing, including the biopsies.
If any of the current 2b participants or any of the 2a participants have some insight as to how they eventually resolved to enter their sons and families to this difficult study, I would appreciate some insight.
If these kids were not so very young, the decision would be much easier. But taking into account that the extension program prolongs the testing even further means that our little ones will be subjected to so much for possibly 2-3 years or more after the main study ends.
I am having some trouble deciding if the means really justifies the end, which itself is, in the case of this particular study drug, still so unknown.