Prof Makris (hemophilia KOL) this morning in a tweet: | SGMO Message Board Posts

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Msg  133788 of 135050  at  9/21/2019 5:13:27 AM  by

Biotech2050


Prof Makris (hemophilia KOL) this morning in a tweet:

 
"Gene therapy has been sold to patients as "once and done". The real durability issue came up after the Biomarin presentation at ISTH. Being able to retreat enforces the durability issue and I feel it will make patients wait for GeneTherapy 2.0 or later. #hemophilia #haemophilia
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Maurice
@MauriceOnTW
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Replying to @ProfMakris
If #GeneTherapy retreatment starts to appear to be a safe viable possibility what would your view on adoption be? What length of durability between retreatments would you consider acceptable?
 
 Glad you were able to see through Biomarin's data. Having a "patient first" mindset is paramount if #hemophilia #genetherapy is to succeed. Biomarin's agenda to capture the full #haemophilia market immediately (and to hell with patients' best interest):
 
 


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