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We have not taken this decision lightly as we look to protect the research we are invested in as…
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You can read the statement in full here.
The statement sets out considerations for patients (and/…
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What is genome editing?
Genome editing is a technique that allows scientists to change the DNA,…
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AveXis started to roll out this programme in January 2020 for children age under 2 years who have…
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While muscle-wasting conditions affect around 70,000 people in the UK, the conditions MDUK supports…
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After an international study, The IMP has released updated guidance on the medicines that should be…
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The study showed improvement in muscle function in people with SMA type 2 and 3 when treated with…
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The study will involve an interview with a researcher which would take around 1 to 1.5 hours and…
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The teams have identified the molecular target for the utrophin modulator drug, ezutromid. Utrophin…
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The ENMC is funded by several European patient organisations and supported by a company forum (made…