Tofersen approved by the MHRA to treat rare inherited form of motor neurone disease

News Healthcare & Pharma Law & Policy Updates

Date: 28th Jul, 2025 Source: Medicines and Healthcare products Regulatory Agency (MHRA) Country: United Kingdom Original Source

Brief

On July 22, 2025, the Medicines and Healthcare products Regulatory Agency (MHRA) approved tofersen (Qalsody) for adults with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. Tofersen works by reducing toxic protein levels produced by the SOD1 gene, which damages nerve cells in ALS patients.

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Medicines and Healthcare products Regulatory Agency (MHRA)

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Tofersen approved by the MHRA to treat rare inherited form of motor neurone disease

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Tofersen approved by the MHRA to treat rare inherited form of motor neurone disease

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