Does the combination data from the mouse model confirm or dispel...

Does the combination data from the mouse model confirm or dispel the proposition that ATL1102 works synergistically with dystrophin replacement therapies? The answer appears to be affirmative. Apart from the functional outcomes, the modulation of proteins indicate firstly, the reduction of inflammation and secondly, the augmentation of muscle recovery.

The importance of targeting inflammation pathways in DMD was outlined back in August 2015 in the article Immune-mediated pathology in Duchenne muscular dystrophy. If I understand correctly, one of the key contentions in the abstract is that the efficacy of dystrophin replacement therapies will be contingent upon modulating the inflammatory response and inducing immunological tolerance to de nova dystrophin expression. The authors include four members of the Center for Drug Evaluation and Research, US FDA.

The scientific guidance has been evident for some time but as previously noted in another post, it was difficult to be heard above the buzz that micro-dystrophin may be a cure. Now everybody will be listening.