Raxone is an oral medication, authorized at a daily dose of 900 mg (given as 2 tablets three times a day with food), for the treatment of visual impairment in adolescent and adult patients with LHON
These increases reflect the expansion of Santhera's operations, especially the commercial activities, the ongoing roll-out of Raxone for LHON
across Europe and market entry preparations for Raxone for DMD.
Consistently, a recently developed mouse model revealed, together with some of the key histopathological features typically observed in LHON
patients, the decrease of complex I activity, respiratory defects, and the increase in ROS levels, but no reduction in ATP synthesis, pointing to oxidative stress as the major driver of the pathology .
is a mitochondrial disease that is caused by well characterized mutations in mitochondrial DNA (mtDNA), with a prevalence of carriers of about 1 in 300 .
and DMD are orphan indications with no approved therapies and a large overlap of specialists (neurologists) facilitating a fast market entry and ValuationLab assumes EU and US approvals and product sales starting in late 2015/early 2016.
He tested positive for the 11778G>A mitochondrial mutation associated with LHON
8220;We are hopeful patients with LHON
may show visual benefit from the treatment protocols we are offering.
RELEVANCE: In this article, we add to the candidate occupational/environmental triggers of LHON
and highlight the need for appropriate case--control (and laboratory) studies to validate the causal effect of mixed toxic exposures.
The five most frequent, primary mutations responsible for LHON
(3460, 9804, 11778, 14484, 15257 ) are included and the LHON
assay was tested on 47 consecutive patients; for 11 patients the clinical diagnosis was confirmed.
is a heritable genetic disease causing blindness.
Raxone represents a major breakthrough in mitochondrial disease treatment, and its approval paves the way for patients with LHON
to be treated and to achieve a meaningful improvement of their visual acuity," said Thomas Klopstock, MD, Professor for Neurology at the University of Munich, LHON
researcher and coordinator of the German network for mitochondrial disorders, mitoNET.
In addition to the RHODOS study data, the MAA includes nonclinical data from a disease-relevant model of LHON
which show that retinal ganglion cell death and other retinal pathologies induced by mitochondrial complex 1 inhibition, could be prevented with idebenone.