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» Cure Rare Disease’s CRD-003 designated orphan drug for LGMD2i/R9
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Neurology/psychiatric
Cure Rare Disease’s CRD-003 designated orphan drug for LGMD2i/R9
Sep. 25, 2025
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The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic mutations in the FKRP gene.
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Regulatory
Genetic/congenital
Neurology/psychiatric
FDA
Orphan drug