FDA grants orphan drug designation for LHON gene therapy

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Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release. Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release. “[NR082] uses recombinant adeno-associated […]