Kubota Vision published the results of a dose range study of a drug known as Emixustat, with findings that will effect dose selection for their phase III trial set to complete in June 2022.Stem-cell therapy involves injecting cells with the potential to mature into differentiated and functioning retinal cells. This therapy has the potential stop disease progression and in the long term improve vision. To improve vision this technique will need to replicate the complex multi-layered and neurally  anatomy of the retina. There are a number of research groups working with stem cells one of which is Ocata Therapeutics.Alkeus Pharma is evaluating the potential of deuterated vitamin A as the drug ALK-001. The hope is that the deuterated vitamin A will reduce the build-up of toxic vitamin A metabolites in the retina and therefore slow rate of visual deterioration. To create deuterated vitamin A some of the hydrogen atoms are replaced with the isotope  deuterium  which has an extra neutron and is therefore twice the standard atomic weight of hydrogen. A Phase II clinical trial is taking place using ALK-001 with an estimated completion date of December 2024.MD Stem Cells, a research-physician clinical development company using autologous bone marrow derived stem cells (BMSC), has released results of the Stargardt Disease cohort within their ongoing Stem Cell Ophthalmology Study II (SCOTS2) clinical trial (NCT 03011541).
On the other hand, the consumption of oily fish, in a diet similar to that which doctors recommend for age related macular degeneration, can be used to slow the progression of the disease.Advances in technology have brought devices that help Stargardt patients who are losing their vision maintain their independence. Low-vision aids can range from hand lenses to electronic devices and can allow those losing their vision to be able to carry out daily activities. Some patients may even opt for in-person services. Prognosis
The long-term prognosis for patients with Stargardt disease is widely variable and depends on the age of onset and genetic alleles.The majority of patients will progress to legal blindness, which means that central reading vision will be lost. However, perimetry and microperimetry studies indicate that the peripheral light sensitivity is preserved over a long time in a significant fraction of all patients (i.e., >50%). Stargardt disease has no impact on general health and life expectancy is normal. Some patients, usually those with the late-onset form, can maintain excellent visual acuities for extended periods and are therefore able to perform tasks such as reading or driving. Epidemiology
A 2017 prospective epidemiologic study that recruited 81 patients with STGD over 12 months reported an incidence of between 1 and 1.28 per 10 000 individuals. The median age of presentation was 27 years (range 5–64 years), most (90%) were symptomatic, with a median visual acuity of Snellen equivalent 20/66. History
Karl Stargardt (1875–1927) was a German ophthalmologist born in Berlin.