Stargardt eye disease, also known as Stargardt degeneration, usually manifests before age 20 and causes progressive loss of central vision and color perception. It’s usually an irreversible condition.

LBS-008, an oral drug approved in both the US and EU as an orphan drug, may help prevent and alleviate Stargardt disease by inhibiting toxin accumulation. It recently earned orphan drug designation.

1. Stem Cell Treatment

Mutations in the ABCA4 gene cause Stargardt disease by gradually damaging retinal pigment epithelium (RPE), which protects and maintains photoreceptor cells, until vision gradually declines until it’s lost completely. Traditional treatments may temporarily delay vision deterioration but cannot restore or stop further loss; stem cell therapy could potentially slow or stop its progress and restore vision at an earlier stage of disease progression.

Embryonic stem cells are master cells, capable of differentiating into any of the various cell types in our bodies. When pluripotent stem cells cultured from patients’ own skin or blood are cultured for use as pluripotent cells, scientists can reprogram them into whatever role is necessary and inject the cells directly into damaged areas or replace aged and dying ones for injection into eye. This has shown great promise in treating numerous ophthalmic conditions including age-related macular degeneration and Retinitis Pigmentosa.

Scientists are also exploring the use of induced pluripotent stem cells (iPS), which are created from somatic cells reprogrammed with pluripotency factors to become pluripotent, as a potential therapy against Stargardt disease and macular degeneration. RPE cells play an essential role in transmitting light signals between eye and brain; by replacing defective RPE cells this therapy may restore vision loss associated with Stargardt disease and macular degeneration.

Clinical trials utilizing human embryonic stem cells have been completed, and results suggest they could be effective treatments. A few Stargardt patients with preoperative 20/400 vision saw improvement to 20/20 or better after receiving bone marrow stem cell transplants; however, this did not reach full macular function recovery; more severe forms of Stargardt didn’t go beyond 20/100 improvement levels.

Researchers used a genetically engineered virus to deliver human retinal pigment epithelium (RPE) cells from human iPS cells into animal models of ABCA4-related macular degeneration. They observed less damage and retained more RPE tissue, leading them to believe iPS cells could offer therapeutic potential against dry AMD.

2. Laser Treatment

Stargardt disease is an inherited form of macular degeneration that attacks the macula – a small area in the center of the retina that allows for clear central vision and facilitates reading and driving. Typically starting during childhood or adolescence, Stargardt gradually worsens central and color vision loss until reading becomes difficult, blurry vision develops, faces become indistinct or colors no longer discernible. Caused by mutations to ABCA4 genes which stop passing essential nutrients to retinal cells resulting in buildups of toxic lipofuscin which ultimately kills retinal cells over time.

Diagnostic of this condition typically involves physical and eye exams in which doctors look for signs of fatty buildup under the macula (yellow deposits), red spots on retina, ocular fluid accumulation (red spots on retina), ineffective cones (yellowish pigments), as well as genetic mutations. A blood sample can further confirm diagnosis.

Although no cure exists for Alzheimer’s Disease, treatment options exist that can improve quality of life and delay progression of symptoms. These can include dietary supplements like saffron and docosahexaenoic acid as well as pharmaceutical treatments like Emuxistat Hydrochloride which is currently under development by Nanoscope Therapeutics.

This company specializes in gene-agnostic optogenetic therapies to treat millions of patients blinded by retinal degenerative diseases for which no treatments exist. Their lead product MCO-010 is currently in a Phase 2b randomized controlled trial for retinitis pigmentosa and has orphan drug status for Stargardt disease and geographic atrophy.

This case study of a Stargardt disease patient treated with dilute echothiophate iodide provides insight into its efficacy for improving her quality of vision, including BCVA and color vision. Furthermore, results of treatment indicate its potential to decrease disability from various retinal conditions including dry age-related macular degeneration and retinitis pigmentosa; additionally this therapy could potentially decrease vascular anomalies and scarring that interfere with vision under these circumstances.

3. Low Vision Aids

Stargardt disease is an inherited disorder affecting central vision of both eyes. Also referred to as Stargardt Macular Dystrophy or Juvenile Macular Degeneration, this condition typically manifests itself during childhood or adolescence and results in gradual deterioration of both central portions, leading to loss of visual acuity and color vision over time.

Stargardt’s disease cannot be cured, but lifestyle modifications may slow its progress. One effective strategy for doing this is wearing sunglasses and wide-brimmed hats when outdoors during daytime hours to protect eyes from UV radiation that can cause cataracts or macular degeneration. Also recommended: eating a diet rich in fruits and vegetables for maintaining good vision.

Doctors conduct comprehensive eye exams to accurately diagnose this condition. This involves inspecting the retina, performing tests such as visual acuity and Amsler grid visual tests and fluorescein angiography to detect any leakage of vessels in the macula, among other measures.

Low vision aids may help people cope with this condition and enable them to function as normally as possible, including magnifying glasses, telescopic lenses and electronic devices.

An alternative approach may involve low-dose medication like echothiophate iodide. This medication can help improve vision by increasing retinal levels of acetylcholine, an essential molecule necessary for photoreceptor cell health and regulation by the enzyme acetylcholinesterase; echothiophate iodide inhibits this enzyme so as to allow more acetylcholine production within your body.

If your child is showing any symptoms of Stargardt’s disease, it is advisable to arrange an appointment with an ophthalmologist as soon as possible. An ophthalmologist will be able to diagnose their condition and suggest what low vision aids may best meet your child’s needs; low-tech solutions like magnifying glasses and telescopic lenses may be purchased at most medical supply stores while larger, more advanced devices may be purchased through mail order catalog companies.

4. Eyeglasses

Eyeglasses are one of the most widely-used low-vision aids. Aside from corrective eye surgery, they’re usually prescribed with a prescription that includes lens power or strength as well as cylinder and axis values to address each individual patient’s refractive error. Glasses may help treat Stargardt disease as well as conditions such as dry age-related macular degeneration, glaucoma and proliferative vitreoretinopathy caused by retinal detachment – just to name a few.

Eyeglasses are meant to be worn over the eyes to provide magnification, improving close-up vision. There are various shapes and sizes of glasses designed to meet individual needs; plastic lenses such as those known by their abbreviations CR-39, polycarbonate, or acetate are the most widely available and provide good optical qualities while being available in an assortment of colors and frame styles or shapes. Polycarbonate offers lightweight durability but poor optics due to a low Abbe number while Acetate allows flexibility while having high abbreviations abbreviation for polarization abbreviation numbers; Acetate offers flexibility with more colors offered options while Acetate stands out due its high abbreviations for polarization abbreviation abbreviation for polarization abbreviation while CR-39 lenses are widely popular because they provide good optical quality lenses which can fit into virtually all frame styles or shapes available on the market today.

Stargardt disease often presents itself with central vision that has become distorted or blurry, often worse in one eye than the other. Color perception may also be affected, with edges of objects seeming wavy or washed-out; all these symptoms can interfere with daily activities and in some instances result in legal blindness.

Low-vision aids can assist patients in managing symptoms and maintaining independence. Aside from wearing glasses, such as electronic magnifiers and bioptic telescopic glasses are also helpful tools in managing low-vision. In addition, eating a diet rich in fruits and vegetables may slow the progression of disease progression.

Ethiophate Iodide (ETI) provided early treatment has significantly enhanced visual acuity and color vision in a Stargardt disease patient with mild myopia, according to this case study published in JAMA Ophthalmology. His best corrected distance and near acuities were measured using ETDRS charts at 50 cm with or without wearable magnifiers, Octopus visual field perimetry tests, and multi-luminance shape discrimination tests.