Public articles linked to the same research event.
Nature News A man with a slowly progressing form of motor neuron disease (ALS) caused by a rare CHCHD10 mutation became the first person to receive an RNA antisense oligonucleotide therapy targeting his specific disease-causing mutation; after three 50-milligram and three 75-milligram doses delivered into his spine between April 2024 and April 2025, he had no serious side effects, and one year later his blood neurofilament light chain levels had fallen to the normal reference range, his motor, breathing and neurological function scores had improved, breathing and cognition scores remained stable, and he continued to work as a physician.
A man with a slowly progressing form of motor neuron disease (ALS) caused by a rare CHCHD10 mutation became the first person to receive an RNA antisense oligonucleotide therapy targeting his specific disease-causing mutation; after three 50-milligram and three 75-milligram doses delivered into his spine between April 2024 and April 2025, he had no serious side effects, and one year later his blood neurofilament light chain levels had fallen to the normal reference range, his motor, breathing and neurological function scores had improved, breathing and cognition scores remained stable, and he continued to work as a physician.
A man with a slowly progressing form of motor neuron disease (ALS) caused by a rare CHCHD10 mutation became the first person to receive an RNA antisense oligonucleotide therapy targeting his specific disease-causing mutation; after three 50-milligram and three 75-milligram doses delivered into his spine between April 2024 and April 2025, he had no serious side effects, and one year later his blood neurofilament light chain levels had fallen to the normal reference range, his motor, breathing and neurological function scores had improved, breathing and cognition scores remained stable, and he continued to work as a physician.
A man with a slowly progressing form of motor neuron disease (ALS) caused by a rare CHCHD10 mutation became the first person to receive an RNA antisense oligonucleotide therapy targeting his specific disease-causing mutation; after three 50-milligram and three 75-milligram doses delivered into his spine between April 2024 and April 2025, he had no serious side effects, and one year later his blood neurofilament light chain levels had fallen to the normal reference range, his motor, breathing and neurological function scores had improved, breathing and cognition scores remained stable, and he continued to work as a physician.