This disappointing result was unexpected based on preclinical studies undertaken by Ionis Pharmaceuticals who, along with the Center, has championed the use of antisense therapeutics for treating neurologic disorders. Click here for more information.
Research undertaken by Dr. Smith on an antisense oligonucleotide clinical trial, published in Lancet Neurology in 2013, was referenced in an article in Science titled "RNA-targeted drugs for neuromuscular diseases" (Vol. 371, Issue 6524, pp. 29-31).