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  1. The drug, which is known scientifically as tofersen and will be sold under the brand name Qalsody, targets a mutation in a gene known as SOD1 that is present in about 2 percent of the roughly 6,000 cases of A.L.S. diagnosed in the United States each year. Fewer than 500 people in the United States at any given time are expected to be eligible.
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  2. FDA Approves Biogen’s Drug for Rare Form of ALS - The New York …

  3. FDA Grants Accelerated Approval for QALSODY™ (tofersen) for