News FDA reviewers slam PTC's Duchenne drug ahead of meeting FDA staff not persuaded by patchy clinical trial evidence for Translarna.
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 UK's Mission divests kidney drug to Dimerix for $292m Mission Therapeutics has struck a deal to out-license a drug for acute kidney injury to Australia's Dimerix, sharpening its focus on CNS diseases.
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