Blog
RSS FeedPosted by Michael Wonder on 26 Feb 2025
Exagamglogene autotemcel for the treatment of patients with severe sickle cell disease
26 February 2025 - NICE has published final evidence-based recommendations on the use of exagamglogene autotemcel (Casgevy) for the treatment ...
Posted by Michael Wonder on 01 Feb 2025
Groundbreaking one-off gene therapy approved for severe sickle cell disease
31 January 2025 - People in England with severe sickle cell disease will be among the first to receive treatment using ...
Posted by Michael Wonder on 31 Jan 2025
Vertex announces Casgevy reimbursement agreement for the treatment of sickle cell disease in England
31 January 2025 - Agreement means eligible sickle cell disease patients in England now have access to Casgevy. ...
Posted by Michael Wonder on 27 Dec 2024
Alnylam Canada signs letter of intent from pan-Canadian Pharmaceutical Alliance for the public reimbursement of Amvuttra for the treatment of hereditary transthyretin-mediated amyloidosis in adults
18 October 2024 - One step closer to achieving public reimbursement of Amvuttra for hereditary transthyretin-mediated amyloidosis. patients in Canada ...
Posted by Michael Wonder on 14 Nov 2024
Qalsody: HAS update
8 November 2024 - On 10 October, the High Authority for Health (HAS) issued a negative decision on the request for ...
Posted 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 09 Aug 2024
BioMarin announces updated strategy for Roctavian to focus on US, Germany and Italy
5 August 2024 - Strategic focus will reduce operating expenses with the goal of achieving Roctavian profitability by end of 2025. ...
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 06 Aug 2024
PBS listing for Australians with hereditary transthyretin-mediated amyloidosis with polyneuropathy
1 August 2024 - Medison Pharma, alongside their partner Alnylam Pharmaceuticals, are pleased to announce that from 1 August 2024, ...
Posted by Michael Wonder on 17 Jul 2024
CSL Behring announces positive reimbursement decision in Canada for Hemgenix (etranacogene dezaparvovec), the first gene therapy for haemophilia B
16 July 2024 - The positive decision from CADTH recognises the importance of this one-time, single dose innovative treatment for ...
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 05 Jul 2024
CSL Behring announces first two patients treated with Hemgenix (etranacogene dezaparvovec) gene therapy for haemophilia B in Europe
4 July 2024 - CSL Behring today announced that two haemophilia B patients were treated with the gene therapy Hemgenix ...
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 ...