See all the latest news, blogs and updates from the medical sector.
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SMC accepted pegungalsidase alfa (Elfabrio®) for the long-term treatment of adults with Fabry disease
The Scottish Medicines Consortium (SMC) has accepted pegungalsidase alfa (Elfabrio®), for the long-term treatment of adults with Fabry disease for restricted use.
08/07/2024
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Update for patients with late onset LAL D (Lysosomal acid lipase deficiency)
Despite best efforts, NICE was unable to make a recommendation for the use of sebelipase alfa in LAL-D patients with non-Wolman disease.
26/06/2024
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Study to create a physical activity intervention for adults with Fabry disease
Get involved in a research study to help people with Fabry disease get more active and improve their mental health and quality of life.
06/02/2024
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NICE approves life-saving treatment for children with infantile lysosomal acid lipase deficiency
The MPS Society is delighted to announce that sebelipase alfa (Kanuma®) has been recommended by NICE as an option for long-term enzyme replacement therapy for people with infantile lysosomal acid lipase deficiency.
27/11/2023
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SMC do not recommend pegunigalsidase alfa (Elfabrio®) for adult Fabry patients
SMC do not recommend pegunigalsidase alfa (Elfabrio®) for adult Fabry patients.
13/11/2023
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NICE approves life-changing medication for treatment of alpha mannosidosis
NICE have recommended velmanase alfa as an option for treating alpha mannosidosis, if treatment is started in people under 18 years (it can be continued in people who turn 18 while on treatment).
10/11/2023
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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.
