Rare diseases

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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

EU regulators reject Santhera's Raxone for DMD

Swiss company to appeal regulators' decision.

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Innovative Regulatory Pathways Summit

January 29-30, 2018

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

London, UK - January 8, 2015: City of London panorama includes high street with office and governments buildings

Orchard’s gene therapy gains UK ‘promising innovative medici...

A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.

Alnylam soars after drug success, paving way for RNAi era

EU regulators reject Santhera's Raxone for DMD

Innovative Regulatory Pathways Summit

FDA's Gottlieb vows to close orphan drug loopholes

Alnylam halts trials of haemophilia drug after patient death

Orchard’s gene therapy gains UK ‘promising innovative medici...

GBL joins CVC in €10.7bn takeover bid for Recordati

Discover a new era of healthcare panel engagement: Grounded ...

Rare diseases

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