New Methods To Evaluate Stargardt Disease Treatments

Vision loss is a major symptom of Stargardt disease, so a main goal of treatments is to combat this loss. In order to test the effectiveness of these treatments, there needs to be a method to track disease progression. The Institute of Molecular and Clinical Ophthalmology Basel and University of Basel have found that microperimetry testing may be a way to see if therapies can slow the progression of vision loss.

About Stargardt Disease

Stargardt disease affects the retina, usually resulting in vision loss. It typically affects children and adolescents, although mild cases may not notice symptoms until early adulthood. While affected individuals do not typically reach total blindness, they often lose their vision to the point of 20/200 or worse. They also experience color blindness, the loss of peripheral vision, delayed adjustment to changes in light, spots in the center of their vision, and sensitivity to bright light. These symptoms are most often caused by a mutated ABCA4 gene.

A diagnosis can be reached through a variety of tests, but doctors will start with an examination of the retina. Other tests include OCTs, ERGs, color testing, Fundus photos, and visual field testing. After a diagnosis is obtained, treatment consists of visual aids and living healthy lifestyles. There are no treatments or cures for Stargardt disease.

New Ways to Test the Efficacy of Treatments

A study, called the ProgStar study, that has been published in JAMA Ophthalmology has shown that microperimetry testing should be used to evaluate the efficacy of novel treatments. This form of testing is able to measure structural changes in the retina, along with functional loss of vision. In fact, microperimetry testing is able to evaluate functional deficits before other testing methods are. The abilities of this method have the ability to see vision loss before the structure of the retina changes, allowing early intervention in terms of treatment.

Because of the abilities of microperimetry testing, it should be used to evaluate novel treatments in clinical trials. The Institute of Molecular and Clinical Ophthalmology Basel, who created the study of this testing, actually plans to use it to evaluate their own gene therapy for Stargardt disease. It should be tested within the next five years. Hopefully this new method of testing and gene therapy will be viable options for this condition.

Read more about microperimetry testing here.


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