Spruce Biosciences (SPRB) said late Monday its long-term data for tralesinidase alfa enzyme replacement therapy in patients with fatal genetic disease Sanfilippo Syndrome Type B or MPS IIIB, demonstrated a rapid and durable reduction of heparan sulfate non-reducing end, which is a surrogate endpoint.
Data also showed relatively preserved cognitive and non-cognitive outcomes, including communication and motor skills, over a six-year period, according to a statement.
These findings support the therapy's potential as the first disease-modifying treatment option, the company said.
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