Posted by Michael Wonder on 29 Jul 2025
Tofersen approved by the MHRA to treat rare inherited form of motor neurone disease
28 July 2025 - New genetic therapy approved for SOD1-ALS brings targeted treatment option to patients in the UK.
The MHRA approved tofersen (Qalsody) on 22 July 2025 to treat adults with amyotrophic lateral sclerosis caused by mutations in the SOD1 (an enzyme called superoxide dismutase 1) gene – a rare, inherited form of motor neurone disease.
