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Our fight for the world s most expensive drug that could transform our son s life

‘Our fight for the world’s most expensive drug that could transform our son s life’ Edward was diagnosed at seven weeks with spinal muscular atrophy. His parents are begging authorities for him to get a £1.7m wonder drug Megan Willis, pictured in her garden in Essex with her seven-month-old son, Edward Credit:  Daniel Jones Megan Willis was about eight months into her pregnancy when she noticed that her unborn baby was moving a little less than usual. She saw her doctor, who told her that everything looked fine. Willis assumed she was “being paranoid”, and comforted herself with the old wives’ tale that babies become less fidgety as the mother prepares for birth. “I wasn’t panicking, I didn’t think anything was wrong with him,” remembers Willis, 29, an events manager from Essex. Nor did her partner, John Hall, a retail manager.

Alexion and NICE agree confidential discount for Ultomiris use on the NHS

Alexion and NICE agree confidential discount for Ultomiris use on the NHS NICE said clinical evidence shows Ultomiris is as effective and safe as Alexion s older PNH drug Soliris Alexion and the UK’s National Institute for Health and Care Excellence (NICE) have agreed a confidence commercial discount for the paroxysmal nocturnal haemoglobinuria (PNH) treatment Ultomiris for use within the NHS. The cost-effectiveness watchdog has backed the use of Ultomiris (ravulizumab) to treat PNH in adults with haemolysis with clinical symptoms suggesting high disease activity, or whose disease is clinically stable after receiving Alexion’s older PNH drug Soliris (eculizumab) for six months or more.

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