A break­through for a rare ill­ness

BRINEURA BIOMARIN

Popular Science - - BEST OF WHAT’S NEW 2017 -

Bat­ten dis­ease is one of a group of rare ge­netic dis­or­ders in which de­fec­tive en­zymes in the brain cause se­verely im­paired neu­ro­log­i­cal func­tion­ing. Re­place­ment en­zymes had been too big to cross through the brain’s pro­tec­tive mem­brane. With Brineura, sur­geons by­pass that hur­dle by in­sert­ing a port into a child’s head and in­fus­ing the en­zyme. The tech­nique may be used to treat many sim­i­lar dis­or­ders.

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