In a snap­shot of its rare dis­ease da­ta, Ul­tragenyx boosts case for new sur­ro­gate bio­mark­er

Ul­tragenyx took a step to­ward es­tab­lish­ing a po­ten­tial new sur­ro­gate bio­mark­er Tues­day morn­ing, pre­sent­ing da­ta for a rare dis­ease with no ap­proved treat­ments.

Re­searchers are study­ing whether an AAV gene ther­a­py can treat San­fil­ip­po syn­drome type A (MPS II­IA) by re­duc­ing the buildup of cer­tain car­bo­hy­drates in the brain and cere­brospinal flu­id. The dis­ease stems from a ge­net­ic de­fect caus­ing the body to be un­able to break down and re­cy­cle the car­bo­hy­drate, lead­ing to neu­rode­vel­op­men­tal and phys­i­cal de­cline ear­ly in child­hood and even­tu­al­ly death, usu­al­ly be­fore in­di­vid­u­als turn 20.

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