PRESENTATION · Apr 2025 · PDF
Science
Explore conditions, clinical trials, and the latest research.
These pages contain non-exhaustive scientific information about uses of products that have not yet been approved by regulatory authorities, as their efficacy and safety for these uses have not yet been established. The information is provided for educational and scientific exchange purposes, and does not suggest products should be used in a manner not consistent with their respective label approved by the regulatory authorities, which may differ by geography. Any publication reflects the state of knowledge at the time it was published. The information is not medical advice and cannot replace the independent medical judgement of a healthcare professional.
PUBLICATION · Jan 2025
Human Gene Therapy, Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease
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Canavan GTx BBP-812 - ESGCT 2024 - Positive Data from High Dose Cohort of Phase 1/2 CANaspire Study
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