R&D SpringWorks spins out of Pfizer on rare disease mission A new biotech spun out of Pfizer is to develop four drugs for rare and serious diseases.
News Alnylam soars after drug success, paving way for RNAi era Shares in US biotech Alnylam have soared after a late-stage study of its RNA interference drug patisaran met its primary endpoint.
News FDA's Gottlieb vows to close orphan drug loopholes FDA commissioner Scott Gottlieb is continuing his plans to streamline and accelerate the regulators' practices, but now plans to close down at least one glaring loophole in the Orphan Drug
News Alnylam halts trials of haemophilia drug after patient death Trials will resume once safety protocols have been reviewed.
News Ultragenyx to slash costs after Angelman trial failure Ultragenyx's therapy for the rare genetic disorder Angelman syndrome, apazunersen, has flunked a late-stage trial, crashing its share price.
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