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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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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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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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Moderna closes on EU approval for combined flu/COVID jab

Moderna's combined vaccine for both flu and COVID-19 has been recommended for approval in the EU, after being knocked back in the US.

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Hernexeos box, two pills, and pill bottle

Boehringer gets FDA's second 'national priority' approval

Boehringer Ingelheim's lung cancer drug Hernexeos has become the second medicine approved after an FDA national priority review.

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US lawmakers take FDA to task over rare diseases record

Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.

New FDA knockback hits uniQure and Huntington patients hard

CHMP backs Ipsen drug for childhood brain cancer

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

Moderna closes on EU approval for combined flu/COVID jab

Boehringer gets FDA's second 'national priority' approval

US lawmakers take FDA to task over rare diseases record

Lilly partners with Hims & Hers on obesity drug sales

Discover a new era of healthcare panel engagement: Grounded ...

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