Patients News | pharmaphorum

Sunday 12 July 2026

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Matt Kapusta

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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Genetic Alliance UK

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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SCA meeting

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.

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Eisai unveils digital platform for kidney cancer patients

Eisai has launched a 'digital destination', called Kompass, that aims to support patients after they have been given a diagnosis of kidney cancer.

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Woman with vitiligo on her face

Joy as NICE changes tack on first NHS vitiligo treatment

NHS patients in England with vitiligo will soon be able to access Incyte's Opzelura, the first drug shown to reverse the skin-lightening disorder.

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FDA maps new regulatory route for ultra-rare diseases

FDA maps new regulatory route for ultra-rare diseases

The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.