News uniQure poleaxed as FDA blocks Huntington's therapy uniQure has revealed that the FDA has said it is not prepared to review its Huntington's gene therapy in what seems to be a change in policy.
News Joy as uniQure Huntington's therapy clears pivotal trial uniQure's gene therapy AMT-130 has cleared a trial in Huntington's, setting up filings and raising the prospect of a first treatment for the disease.
News PTC's Huntington study was positive. Why did its stock fall? In a potentially pivotal trial, PTC Therapeutics' therapy for Huntington's disease met its primary endpoint, but there are questions about the data.
News uniQure rockets on map to market for Huntington's therapy uniQure says it has reached agreement with the FDA on a pathway to accelerated approval for its much-anticipated Huntington's disease gene therapy.
News 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.
News Sage wilts as Huntington's prospect flunks trial Sage Therapeutics' run of bad news has continued with a failed phase 2 trial of dalzanemdor as a treatment for Huntington's disease
News NHS "isn't delivering equitable care for rare diseases" A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
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