Manufacturers submit marketing authorisation application in EU for lenadogene nolparvovec for vision loss due to Leber hereditary optic neuropathy

Evidence for use of lenadogene nolparvovec, a recombinant adeno-associated viral vector gene therapy in this rare mitochondrial genetic disease which results in blindness in most patients, comes from two pivotal Phase-III efficacy studies, RESCUE, and REVERSE.

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Biospace Inc.