Family's Race to Save Toddler with Ultrarare Neurological Disorder.
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.
