@RichardRoma6000 @AlbertF9999 @ByMadeleineA IMO $BMRN can sell it considering what Factor costs in these patients &amp; BIG improvement in QOL even w durability questions...
bigger question IMO is if $SGMO / $PFE can show signs of less decline at similar time points will more patients wait?  Will $PFE update freq to encourage?