[synapticfire]

Another promising gene therapy, close to being submitted to the FDA for approval, has been developed by Ohio State University, Nationwide Children's hospital, and Avexis for the treatment, and possibly cure, of Spinal Muscular Atrophy. https://avexis.com

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/

[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.

[rsync]

"Another promising gene therapy, close to being submitted to the FDA for approval, has been developed by Ohio State University, Nationwide Children's hospital, and Avexis"

I would be very interested to know how the economics of that work out. Is this a classic case of the costs and risks publicized (OSU) and the profits privatized (Avexis) ?

[synapticfire]

I believe with so called "Orphan diseases" the economics of research and eventual market release rarely work out for private companies. I believe it takes a research phase funded by charities, grants, and tax breaks for companies to have any interest. As a family affected by this, whatever gets this to market. However, I am sure someone will make money.