PharmaTher Seeks FDA Orphan Drug Designation for GAMT Deficiency Treatment
PharmaTher submitted a request to the FDA for orphan drug designation for its GAMT deficiency treatment.
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PharmaTher Seeks FDA Orphan Drug and Rare Pediatric Designations
PharmaTher submitted applications for orphan drug and rare pediatric disease designations for 3D-printed creatine monohydrate in GAMT deficiency. The program could qualify for a transferable priority review voucher.