Amyotrophic lateral sclerosis candidate tofersen, an antisense oligonucleotide, showed promise in treating a genetic form of the disease in a Phase I/II clinical trial, according to information presented at the American Academy of Neurology's 71st Annual Meeting in Philadelphia. Seventy patients participated in the trial, and studies with more patients are needed to validate the results, said study author Dr. Timothy Miller of Washington University School of Medicine.
Drug shows promise in treating genetic form of ALS
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