Health
Investigational drug for genetic form of ALS showing early promise, trial indicates – Mirage News
An experimental gene-silencing drug designed for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a clinical trial…
An experimental gene-silencing drug designed for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a clinical trial conducted at the Emory ALS Center and other medical centers around the world.
The trial indicated that the “antisense” drug, known as tofersen, lowers levels of a disease-causing protein in people with a type of ALS caused by mutations in the gene SOD1. The results of the study, published in New England Journal of Medicine, have led to the launch o…
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