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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uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.
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.
Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.
Eisai has launched a 'digital destination', called Kompass, that aims to support patients after they have been given a diagnosis of kidney cancer.
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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