Tofersen, ein neues, von der FDA zugelassenes Medikament für eine genetische Form von ALS, verzögert das Fortschreiten der Symptome und den Tod und führt bei etwa einem Viertel der Teilnehmer zu einer Stabilisierung oder Verbesserung. „Das Fortschreiten der Krankheit zu stoppen und innerhalb von 3 bis 5 Jahren Verbesserungen zu erzielen, ist bei dieser Art von ALS bisher unbekannt.“

New ALS drug stabilizes decline with improved strength, mobility for some

Long-term use of tofersen slows ALS disease progression in people with a genetic subtype, provides hope for treating other forms of ALS

Historically, people with amyotrophic lateral sclerosis (ALS) experience a relentless decline of neurological function that eventually robs them of the ability to move, speak, eat or breathe. Now, researchers from Washington University School of Medicine in St. Louis and collaborators report that long-term use of **tofersen, a new drug approved by the Food and Drug Administration (FDA) for a genetic form of this deadly illness, delays symptom progression and death and in about one-quarter of participants leads to stabilization or improvement**.

The new findings, published Dec. 22 in JAMA Neurology, provide long-term follow-up results from a phase 3 trial of tofersen and its open label extension, both co-led by WashU Medicine, that served as the basis for the FDA’s approval in 2023 of the drug for this rare form of ALS.

“**Stopping disease progression and making improvements over three to five years is unheard of in this type of ALS**,” said first author Timothy M. Miller, MD, PhD, the David Clayson Professor of Neurology at WashU Medicine and co-director of the WashU Medicine ALS Center. “Tofersen shows benefits compared with what we expect to see for these participants, with about 25% of participants experiencing improvement. These results provide hope that we can change the trajectory of this devastating disease, and we are optimistic we can do the same for other forms of ALS.”

For those interested, here’s the link to the peer reviewed journal article:

https://jamanetwork.com/journals/jamaneurology/fullarticle/2843130