Tofersen, marketed under the name Qalsody, is a new treatment for ALS that has now been approved for patients with SOD1-ALS, the second most common cause of ALS, at Karolinska University Hospital. The decision is a long-awaited breakthrough in the field of ALS.
For patients and families, this means that a targeted treatment for a genetically defined form of the disease ALS, amyotrophic lateral sclerosis, is finally becoming available.
"For those of us researching ALS at Karolinska Institutet, this is also a clear example of how research can be translated into clinical benefit. Through our studies of disease mechanisms, genetics, biomarkers and precision medicine, we are working to ensure that more patients gain access to effective and personalised treatments in the future," says Caroline Ingre , physician and researcher at the Department of Clinical Neuroscience , KI.
Caroline Ingre continues:
"Tofersen is an important first step, but developments in ALS research are progressing rapidly and give reason for continued hope."
The decision to approve tofersen as a treatment for patients with ALS and an SOD1 mutation came into effect in June 2026.
