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
Documentation on Stargardt disease 3 is limited primarily due to its rarity and the recent identification of its genetic basis. As this condition affects a small number of individuals, systematic clinical studies have been sparse, resulting in a lack of comprehensive characterization of its clinical features.
To navigate your care effectively, consider consulting a retinal specialist with expertise in inherited retinal diseases. Genetic counseling may also be beneficial to understand the implications of ELOVL4 variants for you and your family. While there are currently no patient organizations specifically for Stargardt disease 3, resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information. Additionally, inquire about participation in any available natural history studies or patient registries.
Currently, there are several orphan drugs in development for Stargardt disease 3, including emixustat and a lentiviral vector containing the human ABCA4 gene. Additionally, there are three active clinical trials aimed at exploring potential treatments and interventions. For more information on these trials, you can visit the ClinicalTrials.gov search page at https://clinicaltrials.gov/search?cond=Stargardt%20disease%203.
Actionable guidance for navigating care for Stargardt disease 3
To navigate your care effectively, consider consulting a retinal specialist with expertise in inherited retinal diseases. Genetic counseling may also be beneficial to understand the implications of ELOVL4 variants for you and your family. While there are currently no patient organizations specifically for Stargardt disease 3, resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can provide valuable information. Additionally, inquire about participation in any available natural history studies or patient registries.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Inheritance patterns describe how genetic conditions are passed from parents to children.
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
Online Mendelian Inheritance in Man
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 — 7 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.