According to NHS England, 16-year-old Thakkar from Watford near London was the first child in the UK to benefit from a pioneering CAR T therapy called tisagenlecleucel (Kymriah) thanks to its Cancer Drugs Fund (CDF)
New NHS drug to help patients suffering blood cancers such as leukaemia and lymphoma walesonline.co.uk - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from walesonline.co.uk Daily Mail and Mail on Sunday newspapers.
AstraZeneca s rare disease unit receives NICE recommendation for Wolman disease therapy in infants pmlive.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from pmlive.com Daily Mail and Mail on Sunday newspapers.
A new fund to fast-track patient access to potentially valuable new medicines may incentivize the pharmaceutical industry to develop high-priced drugs for rare diseases with weak evidence on clinical benefits.
A new fund to fast-track patient access to potentially valuable new medicines may incentivize the pharmaceutical industry to develop high-priced drugs for rare diseases with weak evidence on clinical benefits.