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The FDA has granted orphan drug designation to DTx-1252, an investigational small interfering RNA therapeutic to treat Charcot-Marie-Tooth disease type 1A.According to a DTx Pharma press release, the novel therapy is part of the company’s Fatty Acid Ligand Conjugated OligoNucleotides (FALCON) platform, which uses fatty acids to improve biodistribution and cellular uptake of RNA-based

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United States ,Dtx Pharma ,Heather Biele ,Artie Suckow ,Fatty Acid Ligand Conjugated Oligonucleotides ,

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