Drug Provides Glimmer of Hope for Treating ALS
(LibertySons.org) – Motor neuron disease, or amyotrophic lateral sclerosis (ALS), is a devastating affliction. The ailment results from the death of nerves responsible for taking messages from the brain to the muscles. It is a fatal condition, with people generally only living a couple of years after diagnosis. There are no known treatments or cures. But researchers think they’ve stumbled upon a medication that could change the lives of some ALS patients.
'Truly remarkable' drug helps motor neurone disease – BBC News
Some good news! https://t.co/luiLfbmgQS
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A trial study of 108 individuals published in the New England Journal of Medicine on September 22 revealed a drug that could possibly target specific cases of ALS. The treatment would only work in situations where the disease occurs because of the misproduction of the SOD1 protein. Unfortunately, that is the case in only about 2% of sufferers.
The study used gene silencing, with the drug tofersen stopping DNA from excessively producing SOD1. Patients received the treatment through a series of monthly spinal injections in the lumbar area.
Findings showed the drug helped ease the progression of the disease but did not reverse damage. Some subjects in the study said their symptoms eased and they felt better, so there is potential for this treatment to assist with quality of life.
Despite its limitations, researchers still feel the step is miraculous. This study is the first of 25 trials in which the director of the Neuroscience Institute saw a positive result. Experts hope the findings will lead to other discoveries in treating the multiple forms of ALS.
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