Stargardt disease, also known as Stargardt 1 (STGD1), is an autosomal recessive form of retinal dystrophy that is usually characterized by a progressive loss of central vision associated with irregula...
Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
The documentation for Stargardt disease is limited due to its rarity, affecting approximately 1 in 10,000 individuals. This low prevalence restricts the number of systematic clinical studies that can be conducted. Additionally, the genetic basis of the disease is still being characterized, which contributes to the gaps in clinical understanding.
To navigate your care for Stargardt disease, consider consulting a retinal specialist with experience in hereditary retinal dystrophies. Genetic counseling is also recommended to understand the implications of the identified genes, especially for family planning. You might want to explore participation in natural history studies or clinical trials, which can provide access to cutting-edge research and potential therapies. For additional resources, visit the Genetic and Rare Diseases Information Center at rarediseases.info.nih.gov.
There are currently 28 active clinical trials investigating potential treatments for Stargardt disease. Notable orphan drugs in development include various gene therapies and small molecules aimed at addressing the underlying genetic causes. For more information on ongoing trials, you can visit ClinicalTrials.gov and search for Stargardt disease: https://clinicaltrials.gov/search?cond=Stargardt%20disease.
Actionable guidance for navigating care for Stargardt disease
To navigate your care for Stargardt disease, consider consulting a retinal specialist with experience in hereditary retinal dystrophies. Genetic counseling is also recommended to understand the implications of the identified genes, especially for family planning. You might want to explore participation in natural history studies or clinical trials, which can provide access to cutting-edge research and potential therapies. For additional resources, visit the Genetic and Rare Diseases Information Center at rarediseases.info.nih.gov.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 9 companies have orphan drug designations for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.