RareWays is a research directory. It helps you understand the science. It is not medical advice. Always discuss your care options with your healthcare team.
Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Stargardt Disease: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
[Stargardt disease (ABCA4-asssociated retinopathy): Overview of clinical aspects, classification and differential diagnoses].
Dithmar Sophia et al., Die Ophthalmologie (14 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42873269/
- 2.
[Genetic therapies for Stargardt disease].
Ansari Georg et al., Die Ophthalmologie (8 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42850292/
- 3.
Modern Genetic Technologies for the Treatment of Stargardt Syndrome. Part 1. Non-viral Methods OF Delivery of Therapeutic Constructs
Andrew N. Brovin et al., Ophthalmology in Russia (5 October 2026)
https://doi.org/10.18008/1816-5095-2026-3-512-520
- 4.
Deuterated Vitamin A for Stargardt Disease in TEASE-1 Trial.
Huckfeldt Rachel M et al., JAMA ophthalmology (1 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42690644/
- 5.
Safety and Effects of Gildeuretinol Acetate on Retinal Atrophic Lesions in Stargardt Disease: The TEASE-1 Randomized Clinical Trial.
Kay Christine N et al., JAMA ophthalmology (1 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42690655/
Clinical Trials : Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants
NCT07836452 · Recruiting · Phase 1 · City Therapeutics
Ages 12 to 45
https://clinicaltrials.gov/study/NCT07836452
- 2.
Restoration of Central Vision With PRIMA in Patients With Photoreceptor Degeneration
NCT07266584 · Recruiting · Science Corporation
Ages 18 and over
https://clinicaltrials.gov/study/NCT07266584
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Stargardt Disease
Stargardt disease is the most common inherited macular dystrophy, usually caused by changes in the ABCA4 gene. It damages the central part of the retina, so people gradually lose sharp central vision, often from childhood or early adulthood, while side vision is usually kept. Several treatments aimed at slowing vision loss are being tested in clinical trials.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
Morbus Stargardt (STGD1) is the most common autosomal recessive macular dystrophy and is caused by biallelic variants in the ABCA4 gene located on chromosome 1. More than 1200 disease-associated variants have been described, causing a broad phenotypic spectrum. The hallmark feature is a bilateral, typically slowly progressive visual impairment, although best-corrected visual acuity can be preserved for a long time in patients with foveal sparing. The disease is characterized by lipofuscin accumulation in the retinal pigment epithelium, manifesting as fundus flavimaculatus on funduscopic examination, outer retinal atrophy on optical coherence tomography (OCT) and a characteristic pattern on fundus autofluorescence imaging. While earlier classifications were primarily based on clinical and electrophysiological criteria, current approaches increasingly focus on genotype-phenotype correlations, which can also enable prognostic statements. Severe, early-onset disease courses are predominantly associated with loss of function variants, whereas certain genotypes have been linked to milder and later-onset manifestations. The differential diagnosis remains challenging due to considerable phenotypic overlap with other autosomal-dominant and autosomal-recessive macular dystrophies. Moreover, particularly in late-onset cases, differentiation from geographic atrophy secondary to age-related macular degeneration can be necessary.
Common Questions
What is Stargardt Disease?
Stargardt disease is the most common inherited macular dystrophy, usually caused by changes in the ABCA4 gene. It damages the central part of the retina, so people gradually lose sharp central vision, often from childhood or early adulthood, while side vision is usually kept. Several treatments aimed at slowing vision loss are being tested in clinical trials.
How many clinical trials are available for Stargardt Disease?
RareWays currently indexes 39 clinical trials for Stargardt Disease, of which 13 are actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Stargardt Disease come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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