Pre­ci­sion ther­a­py ap­proach se­cures small biotech $42M haul to com­bat dis­ease that in­spired the Ice Buck­et Chal­lenge

Akin to cys­tic fi­bro­sis (CF), sci­en­tists un­der­stand that cer­tain mu­ta­tions con­tribute to the de­vel­op­ment of the fa­tal neu­ro­log­i­cal dis­or­der amy­otroph­ic lat­er­al scle­ro­sis (ALS). And much …

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