See all the latest news, blogs and updates from the medical sector.
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NICE leans towards a no for the treatment of Alpha Mannosidosis
NICE release interim decision not to recommend Velmanase alfa (Lamzede®) for alpha mannosidosis.
10/11/2022
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Velmanase alfa for Alpha Mannosidosis
We are delighted to announce that velmanase alfa, the ERT treatment for alpha mannosidosis has been made accessible under the Scottish...
20/09/2022
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Fabry experiment trial to improve cardiac outcomes
Fabry disease is a genetic condition which leads to accumulation of fat or “sphingolipid” within the tissues, in particular the heart...
29/06/2022
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NICE provides positive feedback
Following the recent news that Elosulfase alfa (Vimizim) has been recommended by NICE (National Institute for Health and Care Excellence)...
08/06/2022
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Takeda to discontinue their TAK-609 clinical trial
It is with great sadness to announce that last week, Takeda announced that after 8 years, they would not be continuing with their trial...
27/05/2022
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NICE approves life-changing medication for treating MPS IVA Morquio
Elosulfase alfa (Vimizim®) has been recommended by NICE for treating MPS IVA Morquio in people of all ages in England and Wales.
21/03/2022
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NICE final guidance approves life-changing gene therapy for treating MLD
We are delighted to announce that Atidarsagene autotemcel (Libmeldy®) has been recommended as an option by NICE (The National Institute...
04/02/2022
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NICE update on the re-evaluation for the treatment of MPS IVA (Morquio A)
NICE (The National Institute of Clinical Excellence), the drug decision making body in England, have publicly released an update today (2...
02/02/2022
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AVROBIO ends its gene therapy trial for Fabry disease
We are sad to announce that the pharmaceutical company AVROBIO has made the difficult decision to deprioritize its gene therapy trial for...
07/01/2022
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NICE leans towards a no for the treatment of MPS IVA (Morquio A)
NICE (National Institute of Clinical Excellence), the drug decision making body in England, have publicly released their interim decision to
