Jacifusen Benefits Certain FUS-ALS Patients
A study published in The Lancet reports that the experimental therapy jacifusen appears to be safe and may slow or reverse disease progression in some patients with FUS-ALS, a rare and aggressive form of amyotrophic lateral sclerosis caused by FUS gene mutations. The therapy showed promising results, including decreased nerve damage markers and, in one case, significant functional recovery, suggesting potential for delaying disease onset in high-risk individuals. Further research through Phase 3 trials is underway to confirm these findings.