The U.S. Food and Drug Administration has approved the drug tofersen for the treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation of the in the gene SOD1 in an accelerated registration procedure - It is the first drug to target the targeting the genetic cause of ALS and a significant scientific advance in the treatment of the disease.
- The U.S. Food and Drug Administration (FDA) has fast-tracked registration of the drug tofersen based on the reduction of neurofilaments, a marker of neurodegeneration
- Amyotrophic lateral sclerosis associated with a mutation in the gene encoding the enzyme superoxide dismutase 1 (SOD1) is a devastating, fatal-causing ultra-rare genetic form of ALS3-4, with which there are approximately 330 people living in the U.S.
Amyotrophic lateral sclerosis (ALS) is a rare, progressive and fatal neurodegenerative disease that causes the loss of motor neurons in the brain and spinal cord that are responsible for controlling voluntary muscle movements. People with ALS experience muscle weakness and atrophy, resulting in a loss of independence through a gradual loss of the ability to move, speak, eat and eventually breathe. The average life expectancy for people with ALS is three to five years from the onset of symptoms.
Many genes are associated with ALS. Genetic testing helps determine if a ...
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