Claire Diemler's Blog (2)

How to make the FDA move forward?

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…


Added by Claire Diemler on September 10, 2012 at 12:17am — No Comments

A question for parents of children in the 51 skipping trials

Hello PPMD Community, my husband and I are sitting here discussing exon skipping... are there parents out there whose sons are involved in current 51 trials, specifically GSK/Prosensa trials? AVI has been pretty forthcoming with their results but we have a hard time finding info on GSK's.   We are wondering what your experiences have been and if you see benefits in your sons (for those in either trials)?

Added by Claire Diemler on June 25, 2012 at 10:23pm — No Comments

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