Where we at?, page-576

ShareTurkey:


Nah definitely not a write off...they had some really good data in MPS VI....MPS I was very good data as well, see post below this one on MPS I.

MPS VI a few highlights:


The 9-HPT involves manipulating pegs within a pegboard as quickly and accurately as possible, providing a quantitative measure of upper extremity function and fine motor control. Results are reported for both the dominant hand and the non-dominant hand. Participants receiving iPPS in the phase 2 clinical trial demonstrated greater improvement than placebo in the 9-hole peg test from baseline to week 25 on at least one hand.

PGIC:

The childhood health assessment questionnaire (CHAQ) disability index score, where 0 =no disability and 3 = very severe disability, demonstrated a response to treatment. When compared to baseline, the change in disability score for the PPS group was -0.350 versus placebo -0.125, where a minimally clinically important difference for improvements -0.188.


GAGS:

Mozz view - Yes GAGS results here weren't anything special, they were in MPS I, could be ERTS interplay. (MPS I was Bone Marrow Treatment).

At the end of the day, in my view, to progress the MPS program a Global Phase 3 needs to be conducted. PAR will no doubt focus squarely on OA so it doesn't tie up further resources in terms of staff and cash. I think it's prudent to do this and revisit the MPS program with a suitable partner at a later stage.