Silence ALS treats 9 patients' ultra-rare ALS caused by CHCHD10...

Silence ALS treats 9 patients' ultra-rare ALS caused by CHCHD10...

Nine patients with an ultra-rare form of amyotrophic lateral sclerosis (ALS) caused by mutations in the CHCHD10 gene were treated with an experimental antisense oligonucleotide (ASO) that’s designed to silence the disease-causing mutations. The treatment was developed as part of Silence ALS, an initiative that designs ASOs, that is, short strands of genetic material, for carriers of ultra-rare, disease-causing genetic mutations. The effort was b…