In new re­search pa­per, No­var­tis out­lines how an old SMA pro­gram could al­ter the course of Hunt­ing­ton's

As ex­per­i­men­tal treat­ments for Hunt­ing­ton’s dis­ease con­tin­ue to suf­fer set­backs, No­var­tis is out­lin­ing a plan for a re­pur­posed SMA drug it hopes can break phar­ma’s los­ing streak.

In a new pa­per pub­lished to Na­ture Com­mu­ni­ca­tions, No­var­tis sci­en­tists de­tailed how a small mol­e­cule called branaplam low­ers the over­all lev­el of mu­tant gene ex­pressed in Hunt­ing­ton’s mouse brains and in neu­rons tak­en from Hunt­ing­ton’s pa­tients, po­ten­tial­ly help­ing curb dis­ease pro­gres­sion. On top of that, re­searchers say the ther­a­peu­tic ef­fects ap­pear short-last­ing and re­versible, sug­gest­ing the drug may even­tu­al­ly prove safe too.

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