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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