[HTML][HTML] Mitochondrial optic neuropathies–disease mechanisms and therapeutic strategies
P Yu-Wai-Man, PG Griffiths, PF Chinnery - Progress in retinal and eye …, 2011 - Elsevier
Leber hereditary optic neuropathy (LHON) and autosomal-dominant optic atrophy (DOA) are
the two most common inherited optic neuropathies in the general population. Both disorders …
the two most common inherited optic neuropathies in the general population. Both disorders …
Leber hereditary optic neuropathy: current perspectives
C Meyerson, G Van Stavern… - Clinical …, 2015 - Taylor & Francis
Leber hereditary optic neuropathy (LHON) is one of the most common inherited optic
neuropathies causing bilateral central vision loss. The disorder results from point mutations …
neuropathies causing bilateral central vision loss. The disorder results from point mutations …
Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy
P Yu-Wai-Man, NJ Newman, V Carelli… - Science translational …, 2020 - science.org
REVERSE is a randomized, double-masked, sham-controlled, multicenter, phase 3 clinical
trial that evaluated the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects …
trial that evaluated the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects …
[HTML][HTML] Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset
NJ Newman, P Yu-Wai-Man, V Carelli, ML Moster… - Ophthalmology, 2021 - Elsevier
Purpose To evaluate the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects
with visual loss from Leber hereditary optic neuropathy (LHON). Design RESCUE is a …
with visual loss from Leber hereditary optic neuropathy (LHON). Design RESCUE is a …
A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy
T Klopstock, P Yu-Wai-Man, K Dimitriadis, J Rouleau… - Brain, 2011 - academic.oup.com
Major advances in understanding the pathogenesis of inherited metabolic disease caused
by mitochondrial DNA mutations have yet to translate into treatments of proven efficacy …
by mitochondrial DNA mutations have yet to translate into treatments of proven efficacy …
A neurodegenerative perspective on mitochondrial optic neuropathies
Mitochondrial optic neuropathies constitute an important cause of chronic visual morbidity
and registrable blindness in both the paediatric and adult population. It is a genetically …
and registrable blindness in both the paediatric and adult population. It is a genetically …
Coenzyme Q10 as a therapy for mitochondrial disease
IP Hargreaves - The international journal of biochemistry & cell biology, 2014 - Elsevier
Abstract Treatment of mitochondrial respiratory chain (MRC) disorders is extremely difficult,
however, coenzyme Q 10 (CoQ 10) and its synthetic analogues are the only agents which …
however, coenzyme Q 10 (CoQ 10) and its synthetic analogues are the only agents which …
Understanding the molecular basis and pathogenesis of hereditary optic neuropathies: towards improved diagnosis and management
NJ Newman, P Yu-Wai-Man, V Biousse… - The Lancet …, 2023 - thelancet.com
Hereditary optic neuropathies result from defects in the human genome, both nuclear and
mitochondrial. The two main and most recognised phenotypes are dominant optic atrophy …
mitochondrial. The two main and most recognised phenotypes are dominant optic atrophy …
Treatment strategies for inherited optic neuropathies: past, present and future
Bilateral visual loss secondary to inherited optic neuropathies is an important cause of
registrable blindness among children and young adults. The two prototypal disorders seen …
registrable blindness among children and young adults. The two prototypal disorders seen …
Idebenone: a review in Leber's hereditary optic neuropathy
KA Lyseng-Williamson - Drugs, 2016 - Springer
Idebenone (Raxone®), a short-chain benzoquinone, is the only disease-specific drug
approved to treat visual impairment in adolescents and adults with Leber's hereditary optic …
approved to treat visual impairment in adolescents and adults with Leber's hereditary optic …