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Professors Muntoni and Bönneman and their teams have recently jointly published a paper (that the…
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Funding and supporting research remains one of our priorities, and our ultimate goal remains the…
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There are currently no treatments or cures for the condition, which affects an estimated 2,500…
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The group - which Muscular Dystrophy UK is a member of - made the announcement this week.
It…
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As well as campaigning for access to healthcare and treatment and providing direct support to…
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People with SMA Type 1 and 2 may be able to take the oral drug if Spinraza, the first treatment for…
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On 6th May, SMA UK, TreatSMA and MDUK, sent this joint letter to the NICE Programme Director –…
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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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AveXis started to roll out this programme in January 2020 for children age under 2 years who have…