See all the latest news, blogs and updates from the medical sector.
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NICE approves its first ERT for treating Fabry disease
Pegunigalsidase alfa (Elfabrio®) has been recommended as an option by NICE for treating adults with Fabry in England and Wales.
01/09/2023
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Gene therapy clinical trial for patients with MPS II Hunter opens in Manchester
A new gene therapy clinical trial for patients with MPS II Hunter disease is opening in Manchester.
16/06/2023
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NICE unfortunately leans towards a ‘no’ for the treatment of early onset LAL D (Wolman disease)
NICE (National Institute of Clinical Excellence), the drug decision making body, have publicly released their interim decision today not...
05/06/2023
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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...
