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Stargardt’s disease

Stargardt’s disease is the most common inherited macular dystrophy in children with an estimated prevalence of 1 in 8000. It typically presents during the first two decades of life and it often progresses to legal blindness. Understandably, patients who develop the disease at a younger age have poorer visual prognosis than patients with later onset. It is the subject of many clinical and pre-clinical trials with stem cell therapy, gene treatments and various pharmacological approaches being suggested; yet there is no currently approved intervention for preventing or reversing vision loss for these patients. 

The disease is characterized by central vision loss due to premature and progressive accumulation of lipofuscin in the macula, at the level of the retinal pigment epithelium (RPE).  Lipofuscin, commonly known as the ‘wear and tear pigment’, appears as yellow- white autofluorescent flecks in the retina, and it is a common denominator of many retinal dystrophies.  Animal experiments have recently clarified our understanding of the bio-chemical cascade responsible for the excessive and premature accumulation of lipofuscin, and it is now recognized that a reaction with all-trans-retinal (atRAL) forms the byproduct A2PE, which then converts to A2E, the major constituent of lipofuscin in the RPE. 

Ophthalmos study on Stargardt’s mice model
The Stargardts mice model (ABCA4 knockout model) has significantly elevated retinal levels of A2E.We fed the stargardt mice model with Ophthalmos treatment so that their blood AA/EPA ratio was within  Ophthalmos therapeutic zone  daily for 3 months by gavage. 

Results of the study
There were >3 times the amount of A2E in the retinas of stargardt’s mice model compared to the stargardt’s mice model fed for 3 months with Ophthalmos treatment  daily by gavage 
Photos under the electron microscope show >65% reduction of melanosomelipofuscins in the Stargardt’s treated mice compared to the untreated e.g. :

Untreated Stargardt’s mice:                                              Stargardt’s mice treated with daily gavage

56 eyes of 30 patients with Macular dystrophy were given the Ophthalmos treatment. The mean initial visual acuity of patients was 6/18-2, among them 24 eyes with mean initial visual acuity 4% (blind level). Supplementation with Ophthalmos treatment  resulted in significant improvement of the mean visual acuity in all patients by six months . By the six- month time point, the average increase in vision was 15.3 letters . All blind patients went off the blind list within 3-5 months.  

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