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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

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