A characteristic of a disease in which the cause of the disease is a genetic problem inherited from either or both parents.
Comprehensive, easy-to-understand information about this condition
How we create this content →Connect with organizations supporting the inherited community
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Connect with organizations supporting the inherited community
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Connect with organizations supporting the inherited community
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 6, 2026
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
AI-Generated Content: This summary was generated using AI. Always consult with qualified healthcare providers for medical guidance.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
AI-curated news mentioning inherited
Updated Feb 6, 2026
Recent advancements in gene therapy show promise for treating rare diseases such as inborn errors of immunity, metabolism disorders, haemoglobinopathies, and inherited blindness. However, despite successful clinical results, access to these therapies remains limited.