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2017 ; 6
(5
): 6
Nephropedia Template TP
gab.com Text
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English Wikipedia
Towards Treatment of Stargardt Disease: Workshop Organized and Sponsored by the
Foundation Fighting Blindness
#MMPMID28920007
Sears AE
; Bernstein PS
; Cideciyan AV
; Hoyng C
; Charbel Issa P
; Palczewski K
; Rosenfeld PJ
; Sadda S
; Schraermeyer U
; Sparrow JR
; Washington I
; Scholl HPN
Transl Vis Sci Technol
2017[Sep]; 6
(5
): 6
PMID28920007
show ga
Accumulation of fluorescent metabolic byproducts of the visual (retinoid) cycle
is associated with photoreceptor and retinal pigment epithelial cell death in
both Stargardt disease and atrophic (nonneovascular) age-related macular
degeneration (AMD). As a consequence of this observation, small molecular
inhibitors of enzymes in the visual cycle were recently tested in clinical trials
as a strategy to protect the retina and retinal pigment epithelium in patients
with atrophic AMD. To address the clinical translational needs for therapies
aimed at both diseases, a workshop organized by the Foundation Fighting Blindness
was hosted by the Department of Pharmacology at Case Western Reserve University
on February 17, 2017, at the Tinkham Veale University Center, Cleveland, OH, USA.
Invited speakers highlighted recent advances in the understanding of the
pathophysiology of Stargardt disease, in terms of its clinical characterization
and the development of endpoints for clinical trials, and discussed the
comparability of therapeutic strategies between atrophic age-related macular
degeneration (AMD) and Stargardt disease. Investigators speculated that reducing
the concentrations of visual cycle precursor substances and/or their byproducts
may provide valid therapeutic options for the treatment of Stargardt disease.
Here we review the workshop's presentations in the context of published
literature to help shape the aims of ongoing research endeavors and aid the
development of therapies for Stargardt disease.