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rare pediatric disease designation

LEXEO Therapeutics' LX2006 Receives the US FDA's Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia

LEXEO Therapeutics' LX2006 Receives the US FDA's Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreic...

  • July 1, 2021
Taysha Gene Therapies' TSHA-101 Receives the US FDA's Orphan Drug Designation and Rare Pediatric Disease Designation for GM2 Gangliosidosis

Taysha Gene Therapies' TSHA-101 Receives the US FDA's Orphan Drug Designation and Rare Pediatric Disease Designation for GM2 Gangl...

  • August 28, 2020
ProQR's QR-421a Receives FDA's Rare Pediatric Disease Designation for Retinitis Pigmentosa

ProQR's QR-421a Receives FDA's Rare Pediatric Disease Designation for Retinitis Pigmentosa

  • January 31, 2020
Atsena Therapeutics’ ATSN-201 Gains the US FDA’s Rare Pediatric Disease Designation for Treating Retinoschisis

    Atsena Therapeutics’ ATSN-201 Gains the US FDA’s Rare Pediatric Disease Designation for Treating Retinoschisis

    • August 14, 2024
    Actio Biosciences’ ABS-0871 Gains Orphan Drug Designation to Treat Charcot-Marie-Tooth Disease 2C

      Actio Biosciences’ ABS-0871 Gains Orphan Drug Designation to Treat Charcot-Marie-Tooth Disease 2C

      • August 9, 2024
      META Pharmaceuticals’ META-001-PH Receives the US FDA’s Rare Pediatric Disease Designation to Treat Primary Hyperoxaluria

        META Pharmaceuticals’ META-001-PH Receives the US FDA’s Rare Pediatric Disease Designation to Treat Primary Hyperoxaluria

        • August 6, 2024
        ConSynance Therapeutics’ CSTI-500 Receives the US FDA’s Rare Pediatric Disease Designation to Treat Prader-Willi Syndrome

          ConSynance Therapeutics’ CSTI-500 Receives the US FDA’s Rare Pediatric Disease Designation to Treat Prader-Willi Syndrome

          • July 25, 2024
          New Drug Designations - April 2024

            New Drug Designations - April 2024

            • May 30, 2024
            New Drug Designations - March 2024

              New Drug Designations - March 2024

              • April 30, 2024
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