Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

May 15th, 2020|Categories: Alternative splicing, Clinical Development, Delivery, Immune Stimulation, Medicinal Chemistry, Pre-Clinical Research, Ribozyme, Toxicology|

Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. JAMA Neurol. 2020 May 26 Pubmed

Oligonucleotide Therapeutics: New Drugs That Could Stop COVID-19

May 6th, 2020|Categories: Perspectives on Current Science|

In response to the COVID-19 pandemic, scientists around the world have joined together in applying their skills and technologies to fight this global threat. Some of the most promising technologies to fight the coronavirus are based on a new type of drug called oligonucleotide ...

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