14 March 2024 - The Department of Health and Social Care has asked the NICE to produce guidance on the ...
2 August 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
19 April 2023 - NICE has published Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for the treatment of babies aged 12 months ...
19 April 2023 - NICE has published evidence based recommendations on eladocagene exuparvovec (Upstaza) for the treatment of patients 18 ...
23 March 2023 - The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended ...
16 February 2023 - A toddler with a rare inherited condition has become the first child to be treated by ...
15 February 2023 - NICE has published final draft guidance on the use of vutrisiran for the treatment of adults ...
19 January 2023 - Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related amyloidosis by NICE, ...
28 March 2022 - NICE has published evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children. ...
4 February 2022 - Children with a rare, fatal, genetic disorder will be able to benefit from a new one-off treatment ...
4 February 2022 - The NHS has struck a confidential deal for what's thought to be the most expensive drug ...
21 October 2021 - NICE have recommended the use of givosiran, a gene silencing therapy, on the NHS in England, as ...
10 August 2021 - Enthusiasm for the use of outcomes based managed entry agreements to manage uncertainties apparent at the time ...
25 May 2021 - A baby with a fatal genetic condition has been "abandoned" in a "race against time" for ...
4 May 2021 - NICE has today announced that more people with the rare genetic disorder spinal muscular atrophy are to ...