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0.00418702023726452 0.0146545708304257 0.0167480809490579 0.0132588974180043 -0.0209351011863223 -0.00628053035589671 -0.027215631542219 -0.0279134682484298
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Avidity Biosciences Receives FDA Rare Pediatric Disease Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping [Yahoo! ...

Avidity Biosciences, Inc. (RNA) 
Company Research Source: Yahoo! Finance
RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to AOC 1044, the company's investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in people living with mutations amenable to exon 44 skipping (DMD44). AOC 1044 is being assessed in the Phase 1/2 EXPLORE44™ trial for people living with DMD44 and is the first of multiple AOCs the company is developing for DMD. In addition to receiving Rare Pediatric Disease Designation, AOC 1044 has been granted Orphan Designation by the FDA and the European Medicines Agency (EMA), and Fast Track Designation by the FDA. DMD is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein that typically starts at a very young age. Currently, there are no therapies approved targeting exon 44. "We are pleased that the FDA has granted Ra Show less Read more
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