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Ann: Orphan Designation for PBT434 for treatment of MSA, page-8

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  1. 1,078 Posts.
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    The Hong Kong poster by Finkelstein et al has most likely been a part of this application. The conclusion in it was "PBT434 reduced alpha-synuclein aggregation and glial cell inclusions, preserved SN neurons and improved motor function in an animal model of MSA".
    I would think that the complete paper will be published quite soon. It could perhaps give us a bit more info how PBT434 works. The results were very clear, "robust" as was told by Stamler. The other important scientific paper in the application has been the only published PBT434 paper by Finkelstein et al (2017).  I think the corresponding request for orphan drug status is soon also in EU and with that the ratio 5/100000 will gradually get to level of some 40.000 -50.000 possible patients in the world. If the improvement in the symptoms is even in humans "robust" (phase 2)  it may be that gradually almost 50% of these will one day get PBT434 (???). But PBT434 works also in many other diseases, in spite not yet  proven.

 
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