Blog
RSS FeedPosted by Michael Wonder on 15 Feb 2023
Vutrisiran for the treatment of patients with hereditary transthyretin-related amyloidosis
15 February 2023 - NICE has published final draft guidance on the use of vutrisiran for the treatment of adults ...
Posted by Michael Wonder on 19 Jan 2023
NICE backs Alnylam’s Amvuttra for rare form of amyloidosis
19 January 2023 - Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related amyloidosis by NICE, ...
Posted by Michael Wonder on 28 Mar 2022
Atidarsagene autotemcel for treating metachromatic leukodystrophy
28 March 2022 - NICE has published evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children. ...
Posted by Michael Wonder on 04 Feb 2022
First gene therapy for rare genetic neurodegenerative disorder in children, recommended in NICE draft guidance
4 February 2022 - Children with a rare, fatal, genetic disorder will be able to benefit from a new one-off treatment ...
Posted by Michael Wonder on 04 Feb 2022
Libmedly: world's 'most expensive' drug recommended for NHS use
4 February 2022 - The NHS has struck a confidential deal for what's thought to be the most expensive drug ...
Posted by Michael Wonder on 21 Oct 2021
NICE recommends gene silencing therapy for porphyria patients on NHS
21 October 2021 - NICE have recommended the use of givosiran, a gene silencing therapy, on the NHS in England, as ...
Posted by Michael Wonder on 10 Aug 2021
Implementing outcomes based managed entry agreements for rare disease treatments: nusinersen and tisagenlecleucel
10 August 2021 - Enthusiasm for the use of outcomes based managed entry agreements to manage uncertainties apparent at the time ...
Posted by Michael Wonder on 26 May 2021
Colchester baby's parents feel 'abandoned' over £1.7 million drug
25 May 2021 - A baby with a fatal genetic condition has been "abandoned" in a "race against time" for ...
Posted by Michael Wonder on 04 May 2021
NICE announces more people eligible for nusinersen sodium following review of Managed Access Agreement
4 May 2021 - NICE has today announced that more people with the rare genetic disorder spinal muscular atrophy are to ...
Posted by Michael Wonder on 08 Mar 2021
NICE approves life changing gene therapy for treating spinal muscular atrophy
8 March 2021 - A new and potentially curative one-off gene therapy for babies with the rare genetic disorder spinal muscular ...
Posted by Michael Wonder on 17 Feb 2021
bluebird bio ‘baffled’ after NICE rejects beta thalassaemia gene therapy
16 February 2021 - It’s back to the drawing board for bluebird bio and its discussions with NICE, which has ...
Posted by Michael Wonder on 12 Sep 2020
NICE widens its Zolgensma appraisal due to European Marketing Authorisation
11 September 2020 - England’s NICE is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy, according ...
Posted by Michael Wonder on 22 Feb 2020
Health ministers condemn Novartis lottery for Zolgensma, the world’s most expensive drug
12 February 2020 - Novartis has held the first draw to choose four babies who will receive its one-shot treatment ...
Posted by Michael Wonder on 04 Jan 2020
NICE refuses Akcea’s Waylivra
3 January 2020 - NICE has issued an Evaluation Consultation Document, refusing Waylivra (volanesorsen) as a treatment for familial chylomicronaemia ...
Posted by Michael Wonder on 10 Dec 2019
Biotech companies defend prices of one-off gene therapy
9 December 2019 - Latest treatments with price tags as high as $2 million require new financing models. ...