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New FDA knockback hits uniQure and Huntington patients hard

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

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Novo Nordisk plans €432m investment in Irish GLP-1 plant

Novo Nordisk invests in an Irish plant as it prepares for regulatory approvals of its Wegovy pill outside the US.

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After phase 3 clean sweep, Roche plans oral BTK filing in MS

Roche is preparing to file for its oral multiple sclerosis drug fenebrutinib after a third phase 3 win, but could liver toxicity be a stumbling block?

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CHMP backs Ipsen drug for childhood brain cancer

Ipsen's Ojemda is on course to become the first targeted medicine in the EU for some children with a form of brain cancer known as low-grade glioma.

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New biotech Atrium born out of Novartis/Avidity marriage

Atrium Therapeutics has launched with $270m in funding and two preclinical siRNA candidates for rare genetic cardiomyopathies.

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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.

New FDA knockback hits uniQure and Huntington patients hard

Novo Nordisk plans €432m investment in Irish GLP-1 plant

After phase 3 clean sweep, Roche plans oral BTK filing in MS

CHMP backs Ipsen drug for childhood brain cancer

New biotech Atrium born out of Novartis/Avidity marriage

NHS "isn't delivering equitable care for rare diseases"

Lilly bags FDA okay for Wegovy pill rival orforglipron

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