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The First Experimental RNA-Targeting Class Of Drugs Called Antisense Oligonucleotides Show Promise For Neurodegenerative Diseases. Ionis-HTTRx Corrected Huntington’s Defects But Had Limited Efficacy In Phase3; Nusinersen Let SMA Babies Walk. These Drugs May Tackle Alzheimer’s, & ALS, Though Delivery & $750k/Yr Costs For SMA, Pose Hurdles.

This may be a turning point in treating neurodegenerative diseases | Science | The Guardian

https://www.theguardian.com/science/2017/dec/15/this-may-be-a-turning-point-in-treating-neurodegenerative-diseases

Success in trials for Huntington’s and Spinal Muscular Atrophy, raises hopes that diseases such as Alzheimer’s and ALS could be tackled using a new class of drugs