New treatment for rare motor neurone disease recommended for approval

1. März 2024 – EMA has recommended granting a marketing authorisation in the European Union for a new therapy for the treatment of adult patients with amyotrophic lateral sclerosis (ALS), a rare and often fatal disease that causes muscles to become weak and leads to paralysis. Qalsody (tofersen) is indicated for the treatment of adults with ALS, who have a mutation in the superoxide dismutase 1 (SOD1) gene.

In patients with amyotrophic lateral sclerosis (ALS) the nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing increasing loss of muscle function and paralysis of voluntary muscles, including respiratory muscle, which ultimately leads to respiratory failure. ALS is a devastating disorder. The mean survival time with ALS is two to five years.

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