The European Medicines Agency has given the green light to a new therapy to treat adult patients with ALS. This is reported by the ALS League. ALS is a rare and often fatal disease that weakens muscles and leads to paralysis.
The average life expectancy of ALS after diagnosis ranges from two to five years. The exact causes are unknown, but according to the ALS League, both genetic and environmental factors may play a role. A genetic mutation that leads to the production of defective SOD1 enzymes occurs in about 2 percent of people with ALS.
The EMA has now approved a drug for the treatment of ALS. Qalsody targets lower production of the SOD1 protein, thereby reducing neuronal damage.
Better physical skills
For this project, 108 individuals with a confirmed SOD1 gene mutation were closely monitored for 28 weeks. The study results showed that Qalsody reduced so-called ‘plasma neurofilament light chain’ concentrations by about 60 percent. The patients also showed better physical skills.
This step brings access to the medicine one step closer. However, the European Commission still needs to issue a permit to market the drug. Price negotiations will then take place at national level.
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