"We're keen to understand what else the drug was doing. So, we used a proteomics approach... and we looked at 7000 different proteins in the blood. We collected patients’ blood at the end of the study and at various times during the study... So that was the designs, looking at the different protein changes during that period.”
“The most important thing for us is, in the non-ambulant boys at the moment, which is in a phase 2b trial, that we reproduce these results of stabilizing upper limb function by the PUL 2.0 and also strength.The other things are almost like secondary beneficial effects, like maintaining muscle mass and maintaining bone, there will be additional benefits that we can hopefully bring to the treatment.”
“We're seeking to stop the secondary causes of damage to muscles in DMD, which is the inflammation. The primary cause is dystrophin that leads to the loss of a structural protein, but then when you use the muscles, they contract, they get damaged, and then the immune system goes, hang on, this shouldn't be here andit's further damage to them. So we're targeting the secondary cause of immune mediated damage in Duchenne.”
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