This year there was a huge shift towards the downstream effects...

This year there was a huge shift towards the downstream effects and in particular Inflammation, with emphasis on combination therapies @George1972

There are a number of possibilities to explain a perceived shift in tone from gene therapy to combination therapy. One explanation is that clinicians, PPMD and patient families who have invested heavily in gene therapy are moving on to ask how do we make it work.

The science is compelling - inflammation is the next battleground.

Whereas it was previously difficult to be heard above the buzz that micro-dystrophin may be a cure, three factors support the ANP story as we move forward. Firstly, ATL1102 is back in the clinic in what could potentially be a registration trial. Secondly, combination data from a mouse model is being scrutinized to confirm or dispel the thesis that ATL1102 works synergistically with dystrophin replacement therapies. Thirdly, the hope that a point will soon be reached where steroid therapies may no longer be necessary.