New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress
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New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress

Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia…

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New Study Suggests Targeted Next Generation Sequencing Can Aid in Identification of Menkes Disease
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New Study Suggests Targeted Next Generation Sequencing Can Aid in Identification of Menkes Disease

  In a recent press release, biopharmaceutical company Fortress Biotech announced that their study regarding ATP7A targeted next generation DNA sequencing for Menkes disease was published in Molecular Genetics and Metabolism…

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Experimental Treatment for Recurrent Respiratory Papillomatosis Earns Orphan Drug Designation
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Experimental Treatment for Recurrent Respiratory Papillomatosis Earns Orphan Drug Designation

According to a story from PR Newswire, the biotechnology company INOVIO has just announced that the company's investigational product candidate INO-3107 has earned Orphan Drug designation from the US Food…

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