We have been presented with two different options by two equally passionate doctors. One doctor has prescribed weekend-dosing prednisone. She is basing the effectiveness of weekend dosing on…Continue
My son Henry, 5 years old, was diagnosed in December. He thought the bloodwork that we had done would help the doctors fix him so he could play soccer and swim...his words. ( I didn't know why we…Continue
Somewhere on PPMD I found a website that provided results of patient surveys as well as information regarding clinical trials. Does this sound familiar to anyone? I can't for the life of me find it…Continue
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…
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)?