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The original was posted on /r/worldnews by /u/blllrrrrr on 2023-11-18 17:20:30.
World's first gene therapy that could offer relief to thousands of people with sickle cell disease and thalassemia was approved in the U.K.
Submitted 2 years ago by bot@lemmit.online [bot] to worldnews@lemmit.online
https://www.cbsnews.com/news/worlds-first-gene-therapy-sickle-cell-thalassemia-approved-uk/
autotldr@lemmings.world [bot] 2 years ago
This is the best summary I could come up with:
Casgevy — made by the Boston-based Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics — was approved by the agency for the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over.
“The use of the word ‘cure’ in relation to sickle cell disease or thalassemia has, up until now, been incompatible,” she said in a statement, calling the MHRA’s approval of gene therapy “a positive moment in history.”
The new medicine, Casgevy, works by targeting the problematic gene in a patient’s bone marrow stem cells so that the body can make properly functioning hemoglobin.
Britain’s regulator said its decision to authorize the gene therapy for sickle cell disease was based on a study done on 29 patients, of whom 28 reported having no severe pain problems for at least one year after being treated.
Vertex Pharmaceuticals said it had not yet established a price for the treatment in Britain and was working with health authorities “to secure reimbursement and access for eligible patients as quickly as possible.”
Casgevy is currently being reviewed by the U.S. Food and Drug Administration; the agency is expected to make a decision early next month, before considering another sickle cell gene therapy.
The original article contains 760 words, the summary contains 202 words. Saved 73%. I’m a bot and I’m open source!