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 MPS IVA (Morquio A)
NICE (National Institute of Clinical Excellence), the drug decision making body in England, have publicly released their interim decision to
12/11/2021
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NICE unfortunately leans towards a no for the treatment of Metachromatic Leukodystrophy (MLD).
The drug decision-making body NICE (National Institute of Clinical Excellence) has publicly released its interim decision today (9 July...
13/07/2021
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Vimizim update from NICE for MPS IVA (Morquio)
Update 10 June 2021: Dear stakeholders, BioMarin has requested an extension to the timelines for the re-evaluation for elosulfase alfa...
03/06/2021
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New COHERE study for MPS II patients
A study is currently being undertaken by the COHERE team at the Royal Manchester Children’s Hospital. ‘COHERE’ is an abbreviation of Core...
27/05/2021
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Phase I/II clinical study to explore the potential of ST-920 investigational gene therapy to treat
Below is a patient newsletter from biotechnology company Sangamo about their Fabry gene therapy trial. The STAAR Study is Recruiting...
25/05/2021
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Treatment for Metachromatic Leukodystrophy (MLD) approved by the European Commission
MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage
22/12/2020
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AVROBIO announce collaborative research agreement with Manchester university for MPS II gene therapy
AVROBIO announce collaborative research agreement with Manchester university for MPS II gene therapy.
04/10/2020
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REGENXBIO announces expansion of gene therapy programme for the treatment of MPS II
REGENXBIO have released an update on the progress of their investigational gene therapy programme for MPS II Hunters disease.
