Since we last posted the incredible news about the FDA approval of the first treatment for SMA several people have asked some great questions that we'd like to share the answers to...
This is a treatment and not a cure for SMA. It allows the back-up gene(s) the ability to produce more full functioning proteins needed to keep existing motor neurons alive.
The treatment cannot reverse any damage that has already been done. It cannot inspire growth of new neurons either; nor does it increase strength on its own.
The idea behind this treatment is that it could potentially slow down the progression of the disease. It is not designed to stop the progression of the disease.
Many kids during the trials showed marked improvements in motor functioning while some showed less. This tells us the drugs works (or doesn't work) on a case by case basis.
Once we go through the red tape and Ella is taking the treatment will we truly know if this is something that will benefit her...what happens from there is yet to be seen.
We are hopeful that Ella will benefit from this treatment...hopeful that all people with SMA will benefit.
