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Recent advances in SMA research have led to the exciting development of genetic therapies, such as…
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Edasalonexent was a potential treatment for preserving muscle function in patients with Duchenne…
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Marking Becker awareness week (4 – 10th October), we were joined in our BMD seminar by Professor…
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The company will soon be providing charities with more information about what this means for people…
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Marking mitochondrial disease Awareness Week (13 – 19 September), we were joined in our…
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NICE widens scope of its appraisal of Zolgensma This will now be in line with the European…
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There are currently few clinical trials being carried out for the condition, which affects about 2,…
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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…