Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation

DNAJC30 defect: a frequent cause of recessive Leber hereditary optic neuropathy and Leigh syndrome

(2022) Sarah L. Stenton et al.   BRAIN

Leber Hereditary Optic Neuropathy Gene Therapy: Adverse Events and Visual Acuity Results of All Patient Groups

(2022) Byron L. Lam et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

The Impact of Leber Hereditary Optic Neuropathy on the Quality of Life of Patients and Their Relatives: A Qualitative Study

(2022) Benson S. Chen et al.   JOURNAL OF NEURO-OPHTHALMOLOGY

Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy

(2022) Nancy J Newman et al.   BRAIN

Study design and baseline characteristics for the reflect gene therapy trial ofm.11778g>A/ND4-LHON

(2022) Prem S Subramanian et al.   BMJ Open Ophthalmology

Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset

(2021) Nancy J. Newman et al.   OPHTHALMOLOGY

Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study

(2021) Catherine Vignal-Clermont et al.   BIODRUGS

Natural history of patients with Leber hereditary optic neuropathy—results from the REALITY study

(2021) Patrick Yu-Wai-Man et al.   EYE

Impaired complex I repair causes recessive Leber’s hereditary optic neuropathy

(2021) Sarah L. Stenton et al.   JOURNAL OF CLINICAL INVESTIGATION

Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison

(2021) Nancy J. Newman et al.   Frontiers in Neurology

Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates

(2021) David J. Calkins et al.   Molecular Therapy-Methods & Clinical Development

Seven Years Follow-up of Gene Therapy for Leber's Hereditary Optic Neuropathy

(2020) Jiajia Yuan et al.   OPHTHALMOLOGY

Visual Outcomes in Leber Hereditary Optic Neuropathy Patients With the m.11778G>A (MTND4) Mitochondrial DNA Mutation

(2020) Nancy J. Newman et al.   JOURNAL OF NEURO-OPHTHALMOLOGY

Real-World Clinical Experience With Idebenone in the Treatment of Leber Hereditary Optic Neuropathy

(2020) Claudia B. Catarino et al.   JOURNAL OF NEURO-OPHTHALMOLOGY

Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy

(2020) Patrick Yu-Wai-Man et al.   Science Translational Medicine

Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review

(2019) Sarah Goring et al.   BMJ Open

Demographics of a Large International Population of Patients Affected by Leber’s Hereditary Optic Neuropathy

(2019) Lissa Poincenot et al.   OPHTHALMOLOGY

Safety of rAAV2/2- ND4 Gene Therapy for Leber Hereditary Optic Neuropathy

(2018) Catherine Vignal et al.   OPHTHALMOLOGY

International Consensus Statement on the Clinical and Therapeutic Management of Leber Hereditary Optic Neuropathy

(2017) Valerio Carelli et al.   JOURNAL OF NEURO-OPHTHALMOLOGY

Natural History of Conversion of Leber's Hereditary Optic Neuropathy

(2017) Tiffany Jean Hwang et al.   OPHTHALMOLOGY

Gene Therapy for Leber Hereditary Optic Neuropathy

(2017) John Guy et al.   OPHTHALMOLOGY

Visual prognosis better in eyes with less severe reduction of visual acuity one year after onset of Leber hereditary optic neuropathy caused by the 11,778 mutation

(2017) Yukihiko Mashima et al.   BMC Ophthalmology

A neurodegenerative perspective on mitochondrial optic neuropathies

(2016) Patrick Yu-Wai-Man et al.   ACTA NEUROPATHOLOGICA

Evaluation of Leberʼs hereditary optic neuropathy patients prior to a gene therapy clinical trial

(2016) Shuo Yang et al.   MEDICINE

Leber’s hereditary optic neuropathy with late disease onset: clinical and molecular characteristics of 20 patients

(2014) Konstantin Dimitriadis et al.   Orphanet Journal of Rare Diseases

Trial End Points and Natural History in Patients With G11778A Leber Hereditary Optic Neuropathy

(2014) Byron L. Lam et al.   JAMA Ophthalmology

A randomized placebo-controlled trial of idebenone in Leber’s hereditary optic neuropathy

(2011) Thomas Klopstock et al.   BRAIN

Very high penetrance and occurrence of Leber’s hereditary optic neuropathy in a large Han Chinese pedigree carrying the ND4 G11778A mutation

(2010) Xiangtian Zhou et al.   MOLECULAR GENETICS AND METABOLISM

Mitochondrial optic neuropathies – Disease mechanisms and therapeutic strategies

(2010) Patrick Yu-Wai-Man et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Extremely Low Penetrance of Leber's Hereditary Optic Neuropathy in 8 Han Chinese Families Carrying the ND4 G11778A Mutation

(2009) Jia Qu et al.   OPHTHALMOLOGY

Optimized Allotopic Expression of the Human Mitochondrial ND4 Prevents Blindness in a Rat Model of Mitochondrial Dysfunction

(2008) Sami Ellouze et al.   AMERICAN JOURNAL OF HUMAN GENETICS

The optimized allotopic expression of ND1 or ND4 genes restores respiratory chain complex I activity in fibroblasts harboring mutations in these genes

(2008) Crystel Bonnet et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Resolving the clinical acuity categories “hand motion” and “counting fingers” using the Freiburg Visual Acuity Test (FrACT)

(2008) C. Lange et al.   GRAEFES ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Inherited mitochondrial optic neuropathies

(2008) P Yu-Wai-Man et al.   JOURNAL OF MEDICAL GENETICS