All articles tagged with #aso treatment
New data on the antisense oligonucleotide jacifusen (ulefnersen) shows remarkable improvements in two ALS patients with FUS mutations, including functional recovery and disease progression slowing, highlighting potential for early, targeted intervention and ongoing clinical trials.
A family is racing to raise $2.5 million to develop an antisense oligonucleotide (ASO) treatment for their 3-year-old son, Henry, who suffers from alternating hemiplegia of childhood (AHC), a rare neurological disorder that can cause life-threatening seizures and paralysis. The ASO treatment would knock down the toxic impact of a genetic mutation causing dysfunction in Henry's brain. No treatment or cure exists for AHC, which affects one in a million people globally. The family is operating as a biotech company, pushing research, hiring scientists, and working with laboratories to develop the drug from scratch.
The Saladino family is racing to raise $2.5 million to develop an antisense oligonucleotide (ASO) treatment for their 3-year-old son, Henry, who suffers from alternating hemiplegia of childhood (AHC), a rare neurological disorder that can cause life-threatening seizures and paralysis. No treatment or cure exists for AHC, which affects one in a million people globally. The ASO treatment would knock down the toxic impact of a genetic mutation causing dysfunction in Henry's brain. If successful, it could be the first treatment for AHC and pave the way for treating other neurological syndromes caused by mutations in the same gene.