What would it take to make ASO therapies available in more locations?

[corylogan]

My wife and I live in Washington state, and have been working with Seattle Children’s. We’re willing to go to some pretty extreme lengths to support our son, and we don’t know what that looks like yet. We also don’t even know if it would make sense for our son to receive ASO therapy. However, we are currently aware that the only place in the world that’s doing this is at Boston Children’s.

I’m just wondering, hypothetically, so that I can think about it going forward: what would it take to make this kind of therapy available through children’s? What would I have to do? Who would I have to convince?

[DylanVerden]

Hi Cory,

Boston Children’s is actually not the only place in the world working on ASOs. Susannah and Sloane are currently being treated by Dr. Jennifer Bain at Columbia University. In addition, several KAND families have engaged with nlorem through local physicians who have ASO administration capacities.

The most important thing for nlorem is having infrastructure in place for both administering the ASO, and rigorously assessing its safety and benefits. Here is a flow chart we made that describes the general process for families interested in ASO therapy.