Ann: Successful MPSVI Safety Review and PARA OA 008 Update, page-41

This passage has apparently caused a little consternation today.



This really isn't the "fingers crossed" message some are characterising it as. It is an explainer that structural effects are only going to begin being visible at the 6-month point where MRI data on BMEL will be available to be presented. What the announcement is saying, is that Day 56 readout can't show these things yet. Donna provides more colour on this at 37.15 in the video below. The question is posed "If primary endpoints in the biomarkers aren't apparent at Day 56, is there any point in continuing to 6 month, and 12 month readouts?". Donna gives a very strong answer as to why they would be. Essentially, visible structural modifications in the knee joint can and will take time to present themselves.


This video is only a month old, but it looks like some people might benefit from jumping also to 33.45 where Donna is asked "What gives us confidence in the 008 results" or back to 11.40 where Marco states of the 008 study that "We believe it will" show that iPPS has disease modifying properties.