Blog
RSS FeedPosted by Michael Wonder on 30 Oct 2024
Eplontersen for the treatment of patients with hereditary transthyretin related amyloidosis
29 October 2024 - NICE has published final drat guidance on the use of eplontersen for the treatment of patients ...
Posted by Michael Wonder on 11 Sep 2024
Exagamglogene autotemcel for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassaemia
11 September 2024 - NICE has published final evidence-based recommendations on the use of exagamglogene autotemcel (Casgevy) for the treatment ...
Posted by Michael Wonder on 08 Aug 2024
World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England
8 August 2024 - Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time ...
Posted by Michael Wonder on 08 Aug 2024
Vertex announces Casgevy reimbursement agreement for the treatment of transfusion-dependent beta thalassemia in England
7 August 2024 - Eligible transfusion-dependent beta thalassaemia patients in England will be able to access the therapy from today. ...
Posted by Michael Wonder on 09 Jul 2024
A new gene therapy, transformative cystic fibrosis medicines and an obesity jab included in guidance recommended during pre-election period
8 July 2024 - More than 20 pieces of draft and final guidance on a range of medicines and medical conditions ...
Posted by Michael Wonder on 29 Jun 2024
NICE recommends CSL Behring’s haemophilia B gene therapy Hemgenix
28 June 2024 - NICE has recommended CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) for immediate reimbursement by ...
Posted by Michael Wonder on 14 Mar 2024
Exagamglogene autotemcel for the treatment of patients with sickle cell disease
14 March 2024 - The Department of Health and Social Care has asked the NICE to produce guidance on the ...
Posted by Michael Wonder on 02 Aug 2023
Etranacogene dezaparvovec for the treatment of patients with moderately severe or severe haemophilia B
2 August 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
Posted by Michael Wonder on 19 Apr 2023
Onasemnogene abeparvovec for the treatment of patients with presymptomatic spinal muscular atrophy
19 April 2023 - NICE has published Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for the treatment of babies aged 12 months ...
Posted by Michael Wonder on 19 Apr 2023
Eladocagene exuparvovec for the treatment of patients with aromatic L-amino acid decarboxylase deficiency
19 April 2023 - NICE has published evidence based recommendations on eladocagene exuparvovec (Upstaza) for the treatment of patients 18 ...
Posted by Michael Wonder on 25 Mar 2023
NICE recommends life-changing gene therapy for children with ultra rare genetic disorder in final draft guidance
23 March 2023 - The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended ...
Posted by Michael Wonder on 17 Feb 2023
UK’s most expensive drug Libmeldy saved Teddi Shaw, but is too late for her sister
16 February 2023 - A toddler with a rare inherited condition has become the first child to be treated by ...
Posted 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. ...