Concerned Mother Says FDA Might Remove Drug Helping Teen Sons Fight Rare Disease
An experimental drug has been a life saver for two brothers in Chester County but the
Two teenage brothers have a rare genetic disease. Theyve been doing well with this experimental drug for years, but the
Like most brothers, Elliott and
You cant do other stuff your friends can do and stuff. And youre different and stuff like that, Elliott said.
Elliott was diagnosed first, ten years ago, and Henry received his diagnosis a month later.
It was the worst day of our life as parents, their mother, Joanna, said. The dreams you have for your children just go up in smoke. Youre lost. We were struggling to cope we didnt know what to do.
Joanna and her husband Paul were told their children with Duchenne would slowly lose their lives, but there was a ray of hope.
The doctor called us and said, I have the worst possible news but I have the best news in the worst possible case scenario and that theres a drug trial,` Joanna recalled.
The drug Ataluren claims to slow the progression of Duchenne. It quickly gave the Johnson brothers more mobility.
Its a life changer for them,
After school, every day, theyre in the backyard playing with their dog, but their fun days are now in jeopardy.
In rejecting the approval, the
I understand they (the
Because its a rare disease, the scope of the drug trial was limited and while it might not have worked for others, it did for these two brothers.