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Neurophth announced the first patient has been dosed in G.O.L.D. clinical trial for the gene therapy treatment of LHON prnasia
June 29, 2021
Neurophth Biotechnology Ltd., a fully-integrated genetic medicines company developing AAV-delivered gene therapies for the treatment of ocular diseases, announced that the first patient has been dosed in G.O.L.D. clinical trial for the treatment of ...
Chiesi's Raxone recommended for use within NHS Wales pharmatimes
April 29, 2021
Chiesi’s rare disease medicine Raxone has been recommended for use within NHS Wales for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).
Neurophth Announces IND Approval by the NMPA for Leber Hereditary Optic Neuropathy Gene Therapy prnasia
April 02, 2021
Neurophth Biotechnology Ltd., a fully-integrated genomic medicines company developing adeno-associated viral (AAV)-delivered gene therapies for the treatment of ocular diseases, recently announced the Center for Drug Evaluation (CDE) of ...
Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by U.S. FDA prnasia
September 27, 2020
Neurophth Therapeutics, Inc., today announced that its leading candidate, NR082 (rAAV2-ND4, NFS-01 project), was granted an orphan drug designation (ODD) by the U.S. FDA for the treatment of Leber's Hereditary Optic Neuropathy associated with ND4 mutation
Santhera, Chiesi Group Enter Raxone License Agreement contractpharma
May 29, 2019
Chiesi gains rights to Raxone in LHON and all other ophthalmological indications in deal valued at €186 million.
GenSight rebuilds its case for LHON gene therapy, eyeing filing next year fiercebiotech
December 26, 2018
GenSight Biologics has reported new data from its phase 3 program for GS010, its gene therapy for an ultrarare eye disease, sparking another uptick in its share price....
FDA grants orphan drug designation to elamipretide for LHON europeanpharmaceuticalreview
May 02, 2018
The FDA granted Orphan Drug Designation to elamipretide, for the treatment of Leber’s hereditary optic neuropathy…