I know of no other gene therapies, but other treatments are very real, and very close to being approved.
My son is in a trial of nusinersen with Ionis Pharmaceuticals.
The results of this drug are astounding.
20 karma · joined July 15, 2013
If you have not heard of SMA, you should look it up. I had not heard of it until my 2 month old son was diagnosed. http://www.curesma.org/sma/about-sma/
"Second, we have requested that the National Highway Traffic Safety Administration conduct a full investigation as soon as possible into the fire incidents."