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- Neurocrine’s rare-disease drug approved but later linked to deaths and serious side effectsNeurocrine Biosciences received regulatory approval for a rare‑disease therapy after a small clinical trial showed modest efficacy and side effects that were considered manageable. The decision was…
- PTC Therapeutics agrees to pay up to $211 million for Sangamo’s experimental Fabry disease gene therapyPTC Therapeutics announced it will invest up to $211 million to acquire rights to an advanced gene therapy for Fabry disease being developed by Sangamo Therapeutics. The therapy, called ST-920 (is…