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Spruce Biosciences reports long-term TA-ERT data stabilizing cognition and motor skills in MPS IIIB
SPRUCE BIOSCIENCES, INC.
- TA-ERT normalized CSF HS-NRE and stabilized Bayley-III cognitive scores over up to 6 years vs decline in natural history.
- VABS-II analysis showed stabilization of receptive/expressive communication and fine/gross motor skills.
- Sibling case study: treated child at 12.1 years spoke words and self-fed; untreated at 11.7 years was nonverbal and dependent.
- Safety consistent with ICV route; ~6,000 doses administered to 22 patients without new safety signals.
- Spruce plans to advance TA-ERT through BLA submission and potential FDA approval for MPS IIIB.