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Rare Pediatric Disease
Novartis' Iptacopan (LNP023) Receives the US FDA's Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation...
December 16, 2020
ProQR's Sepofarsen Receives FDA's Rare Pediatric Disease Designation to Treat Leber's Congenital Amaurosis 10
October 16, 2019
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