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- Ionis shares tumble over $1 billion as Tryngolza sales forecast cut in halfIonis Pharmaceuticals saw its market value drop by more than $1 billion after it lowered its sales outlook for Tryngolza, a triglyceride‑lowering therapy. The company now expects $50 million in sa…
- FDA's new rare-disease framework faces first test with Capricor's Duchenne therapy decisionThe FDA announced a new "fit-for-purpose" rare disease framework that aims to evaluate therapies based on patient need and realistically attainable evidence. The first real test of the approach wi…