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The power of representation, innovation and trust in rare disease clinical trials

By Carol Pitcher-Towner

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Alnylam’s RNAi therapeutic vutrisiran recommended by CHMP for rare heart disease

The committee has recommended the drug for both wild type and hereditary ATTR-CM

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Alnylam scores EU approval for RNAi drug Givlaari

Company says it will work towards value-based agreements for the drug

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Novartis links up with NHS for speedy access to cholesterol drug inclisiran

Collaboration will see drug offered to patients across the UK via clinical study

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Novartis picks up inclisiran after completion of MedCo acquisition

If approved could become blockbuster cholesterol treatment

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Alnylam’s third RNAi drug clears phase 3 trial in rare kidney disease

Plans to file for approval in US and Europe in early 2020

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Novartis acquires The Medicines Company for $9.7bn following takeover rumours

MedCo's cholesterol lowering inclisiran main attraction in planned acquisition

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FDA clears Alnylam’s second RNAi drug for rare disease

Second drug in the class to be cleared by regulatory authority

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The Medicines Company surges on rumours of Novartis takeover

Buyers interested in company as it prepares to file CV drug inclisiran

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TMC preps filings for inclisiran after final phase 3 trials hit the mark

Less frequent dosing schedules could prove an advantage over rivals

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TMC’s full data for inclisiran impresses at ESC

Marketing applications expected before end of year

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TMC rockets on data for twice-yearly cholesterol drug inclisiran

Shares up 14% following announcement

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