DCCR Tablets for GSD1A Granted Orphan Drug Status
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DCCR Tablets for GSD1A Granted Orphan Drug Status

In a news release from June 2, 2021, biopharmaceutical company Soleno Therapeutics, Inc. ("Soleno") shared an exciting update. The company's investigational treatment, DCCR (diazoxide choline) Extended-Release tablets, received Orphan Drug…

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RSC-57 for Kabuki Syndrome Receives Orphan Drug and Rare Pediatric Disease Designations
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RSC-57 for Kabuki Syndrome Receives Orphan Drug and Rare Pediatric Disease Designations

Just over a week ago, drug discovery and development company Rescindo Therapeutics Inc. ("Rescindo") shared that its drug candidate RSC-57 received both Orphan Drug and Rare Pediatric Disease designations. The…

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Sotatercept for PAH Granted Orphan Designation from EC
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Sotatercept for PAH Granted Orphan Designation from EC

In a recent press release, biopharmaceutical company Acceleron Pharma, Inc. ("Acceleron") announced that its investigational drug candidate sotatercept received Orphan designation from the European Commission (EC) for the treatment of…

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Experimental Stargardt Disease Treatment Earns Orphan Drug and Rare Pediatric Disease Designations
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Experimental Stargardt Disease Treatment Earns Orphan Drug and Rare Pediatric Disease Designations

According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…

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Rett Syndrome Treatment TSHA-102 Received Rare Pediatric Disease and Orphan Drug Designations

Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…

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