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Tofersen, an antisense oligonucleotide (ASO) that selectively targets the genetic driver of disease, is being studied for the potential treatment of ALS in adults with a confirmed superoxide dismut...
Original Article: Biogen Inc.: Biogen to Present New Interim Data from Its Phase 1/2 Clinical Study of Tofersen (BIIB067) for the Potential Treatment of a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS)NEXT ARTICLE
Most human diseases are caused by production of abnormal proteins or malfunctioning proteins. Antisense therapy involves inhibiting production of these proteins. When a gene is known to cause a specific disease and the genetic sequence ...