Ap­plied Ther­a­peu­tics show­cas­es ear­ly da­ta in PhI­II rare sug­ar dis­ease tri­al

Af­ter mul­ti­ple set­backs rang­ing from a short sell­er at­tack to clin­i­cal holds, Ap­plied Ther­a­peu­tics is back at it in an­oth­er sug­ar dis­or­der.

Ap­plied Ther­a­peu­tics rolled out in­ter­im Phase III da­ta Thurs­day morn­ing for AT-007 in SORD de­fi­cien­cy, an in­her­it­ed dis­ease where af­fect­ed peo­ple can­not me­tab­o­lize sor­bitol, a type of sug­ar. The dis­ease, re­cent­ly dis­cov­ered in 2020 and part of a group of hered­i­tary dis­or­ders called Char­cot-Marie-Tooth dis­or­ders, re­sults in ac­cu­mu­la­tion of sor­bitol in the body. That ac­cu­mu­la­tion can cause neu­rons to de­gen­er­ate and lead pa­tients to lose mo­bil­i­ty.

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