PTC Therapeutics

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Novartis pays $1bn upfront for Huntington's drug from PTC

Novartis has made a return to the Huntington's disease stage by licensing an oral mRNA splicer from PTC Therapeutics for $1 billion upfront.

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PTC gets FDA okay for first brain-delivered gene therapy

More than two years after its approval in Europe, PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US.

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EU rules PTC’s Duchenne drug can stay on the market

Going against the advice of its own advisors, the European Commission has said that PTC Therapeutics’ Translarna therapy for Duchenne muscular dystrophy (DMD) should stay

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uniQure craters on Huntington’s gene therapy data

uniQure has presented new results from its phase 1/2 study of its gene therapy for Huntington’s disease that

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PTC slides on failed Friedreich ataxia study

PTC Therapeutics has cued up no fewer than three clinical trial readouts this quarter that could lead to new regulatory filings – but the latest for vatiquinone in inherit

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PTC eyes filings for PKU therapy after phase 3 win

PTC Therapeutics is on track to file for approval of a new therapy for rare disease phenylketonuria after a positive phase 3 readout that could extend its product portfoli

Novartis pays $1bn upfront for Huntington's drug from PTC

PTC gets FDA okay for first brain-delivered gene therapy

EU rules PTC’s Duchenne drug can stay on the market

uniQure craters on Huntington’s gene therapy data

PTC slides on failed Friedreich ataxia study

PTC eyes filings for PKU therapy after phase 3 win

World Without Disease initiative: 2024 update

The AI effect in pharma commercial strategy: From data to re...

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