Alex Zhavoronkov (L) and Jeffrey Rothstein (Insilico/Johns Hopkins)

As ALS pa­tients unite around Amy­lyx, a new pa­per hints at po­ten­tial drug­gable tar­gets

ALS is a de­bil­i­tat­ing, uni­ver­sal­ly fa­tal dis­ease. As mo­tor neu­rons die, pa­tients lose their abil­i­ties to walk, cut their own food, swal­low and even­tu­al­ly, breathe. Most pa­tients die with­in three to five years of symp­tom on­set, and there are few ap­proved treat­ments that on­ly mod­est­ly im­pact func­tion and sur­vival.

Though pa­tients and ad­vo­cates have ral­lied around an ex­per­i­men­tal drug from Amy­lyx Phar­ma­ceu­ti­cals, cur­rent­ly be­ing re­viewed by the FDA, they con­tin­ue to em­pha­size how more work needs to be done. Re­search in­to ALS pathol­o­gy re­mains scant rel­a­tive to oth­er fields, as sci­en­tists have yet to dis­cov­er a con­firmed bio­mark­er that mea­sures pa­tients’ progress and have on­ly iden­ti­fied a hand­ful of ge­net­ic tar­gets im­pli­cat­ed in the dis­ease.

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