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- Arrowhead’s siRNA shows strong tau reduction in early human study, outpacing other BBB platformsArrowhead Pharmaceuticals reported that its investigational siRNA candidate ARO-MAPT produced notable reductions in cerebrospinal fluid tau in a Phase 1/2a trial of healthy volunteers. Doses of 2.…
- FDA unveils new rare disease framework to streamline evidence standards and speed therapiesThe FDA announced a new Rare Disease Framework Initiative aimed at standardizing how scientific evidence is evaluated for rare disease therapies. The framework seeks to apply consistent clinical t…BioSpace · 6d agoRegulatoryPositive