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Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [2] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND) or (in the United States) Lou Gehrig's disease (LGD), is a rare, terminal neurodegenerative disorder that results in the progressive loss of both upper and lower motor neurons that normally control voluntary muscle contraction. [3]
An experimental medication may slow the progression of amyotrophic lateral sclerosis, or ALS, researchers reported Wednesday. The research was supported in part by donations from the Ice Bucket ...
The panel of Food and Drug Administration experts voted 17-1 that drugmaker Brainstorm’s stem cell-based treatment has not been shown effective for patients with the fatal, muscle-wasting ...
6. "Treatments for rare genetic diseases are rapidly being translated to clinics. With close to 40 gene and cell therapies approved and hundreds expected to come down the pipeline, I believe that ...
The ALS Association is an American nonprofit organization that funds global amyotrophic lateral sclerosis (ALS) research, provides care services and programs to people affected by ALS through its nationwide network of clinical care centers, and works with ALS advocates around the country for state and federal policies that serve people living with amyotrophic lateral sclerosis (ALS), also ...
The Food and Drug Administration meets this week to consider approval of an experimental treatment for Lou Gehrig’s disease, the culmination of a yearslong lobbying effort by patients with the ...