Huntington's disease

News

uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.

News

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

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

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