A great Poster. Please enjoy.NOTES:Please note data corresponds...

A great Poster. Please enjoy.



NOTES:
Please note data corresponds to just 3 patients, take that into consideration.

Poster file was too big for me to easily be able to upload it to here so I have screenshotted it in parts...there was also an audio file that was also big (Yes I tried to compress it but was still too big). I believe I might have it in a format you can listen to, see part 2 after this post with instructions!

In case you can't listen to it, I'm hoping it will be included on a link in PAR's website in the next day or so??






Highlights? A number:

1) Very clear and comprehensively laid out poster, includes trial design and various tests employed.


2) Some interesting results sub topics covered, Pharmacokinetics, biomarkers, individual joint ROM (Range of Motion), PROMIS scores (Pain function, fatigue, pain inference, see this reference for more on this PROMIS concept: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5131708/ )


3) I liked the biomarker graphic, showing a down trend overall, yes some flat lines but certainly no increases over course of drug treatment.


4) Safety? "All TEAE's have been deemedmild to moderate and resolved, with no SAEs reported".


5) (My emphasis added):

"All patients had a decrease (improvement) in the MPS I signature oligosaccharide GAG fragments" and further, "...from baseline to week 25, with the greatest improvement seen inpatient 01 01-102, who had a 40% drop from the highest baseline levels".


6) "Patient 01 201, receivingthe high dose (1.5 mg/kg) had improved ROM on all joints assessed".


7) Gait, Stairs, Gowers, Chair (GSGC): "All patients demonstrated overall improvement in the GSGCqualitative global score with improvements ranging from 25% 39%over baseline at week 25".


8) Downtrending pain. (Was also trending down for Pain Intensity, Behaviour and Fatigue).






9) All important mobility? Improved!





10) ...and what did the patients think? (Don't forget this plays a critical role in modern times with FDA, Docs and the Medical associations valuing such feedback):










THE POSTER: