Hope for Treatment with Alkeus Pharmaceutical.
The trial is using oral therapy, once a day, Gildeuretinol.
Stargardt disease is caused by the changes in a gene called ABCA4, which influences the way the body used vitamin A, resulting in damage to the retina which leads to progressive vision loss as our peepers cannot process the disposing of waste vitamin A correctly.
There has been promising results so far halting progression of early stage Stargardt’s Disease.
3 teenagers treated with the drug during the 2 to 6 years of treatment, saw no deterioration of the disease.
This suggests that without treatment they would have experienced sight loss within 2 years, similar to the siblings with the same identical condition experienced.
“We’ve been able to demonstrate the very important effectiveness in late stage – we can’t bring the vision back, the vision damage is permanent, but we can preserve some of the last retina that is still alive,” CEO Leonide Saad told Reuters.

