Regarding the recent articles about Jenny McNary's two sons: Sarepta's Chris Garabedian says:
Parents and patients will be crucial to making that case to the FDA, he says: 'Here are parents who are willing to sign waivers that say, 'I don't care that this drug hasn't been tested fully ... because I know the alternative."
How can we in the PPMD community most effectively make this case? Because of the publicity McNary has received, the public seems to understand the importance of making this drug available to both of her sons. The same is true of every boy who can benefit from 51 skipping. It is also important for those of us with sons with other deletions.
What can we do to make the FDA move? Sign me up. Let's do it.