Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis.

26 , 27 Although the high dose of BIIB078 did not result in clinical benefits (as assessed by ALSFRS‐R, Slow Vital Capacity, Hand‐Held Dynamometry, and the Iowa Oral Pressure Instrument), it showed a trend toward a greater decline compared to the placebo. 26 In WVE‐004 for C9‐ALS/FTD, sustained reductions in poly(GP), a pharmacodynamic biomarker indicative of toxic polydipeptides translated from the expanded GGGGCC repeat, were observed from baseline, with a maximal mean reduction of approximately 50%.

also in 53,322 other papers