Patients News | pharmaphorum

News

Loudspeaker used by a man, graphic art representation

'Dismal' survival demands change in bile duct cancer therapy

International cancer experts have called for big changes to bile duct cancer, which kills three-quarters of patients within a year of diagnosis.

News

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.

News

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.

News

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.

News