[Frozenlock]

As one of the unlucky ones with this disease, I'm very happy to see some advances made in the field. (Tho I've been hearing about promising advances for decades, without ever seeing any concrete result...)

Did you find other organizations working on gene therapy for SMA?

[synapticfire]

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.

[Frozenlock]

Thank you very much!

I contacted the company and am now waiting for a response.