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Tofersen is an antisense drug targeting the enzyme superoxide dismutase 1 (SOD1) that is injected directly into the spinal canal once a month, and is being tested in a form of the ...
Beleaguered Biogen has been handed some encouraging news from the FDA, after the regulator agreed to complete its review of amyotrophic lateral sclerosis (ALS) therapy tofersen within six months.
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The anxious wait for life-changing drug approvalIn June 2023, Mr Thomas started tofersen, as part of an early access programme suitable for people with the SOD1 MND gene - he admitted he had to "battle" to gain access at a hospital in England ...
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