Autosomal dominant optic atrophy and peripheral neuropathy (ADOAPN) is a form of autosomal dominant optic atrophy (ADOA), characterized by progressive and isolated visual loss in the first decade of l...
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 7, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 7, 2026
The limited documentation surrounding ADOAPN is largely due to its rarity, which restricts the scope of systematic clinical studies. With fewer cases reported, comprehensive research and characterization of clinical features are challenging. This can lead to feelings of isolation for patients and families affected by the condition, but ongoing research efforts may provide more clarity in the future.
To navigate your care effectively, consider seeking a neurologist with expertise in hereditary neuropathies, as they may have insights into managing your condition. While there are currently no identified patient organizations, you can access resources from the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov for further information. Participating in clinical trials may also offer opportunities for involvement in research and access to potential treatments, so inquire about eligibility for ongoing studies.
Currently, there is one active clinical trial related to ADOAPN. Additionally, an orphan drug designated for this condition is an 18-mer antisense oligonucleotide complementary to OPA1 pre-mRNA, which is still in development. For more information on ongoing research, you can explore the active clinical trials at ClinicalTrials.gov using this link: https://clinicaltrials.gov/search?cond=autosomal%20dominant%20optic%20atrophy%20and%20peripheral%20neuropathy.
Actionable guidance for navigating care for autosomal dominant optic atrophy and peripheral neuropathy
To navigate your care effectively, consider seeking a neurologist with expertise in hereditary neuropathies, as they may have insights into managing your condition. While there are currently no identified patient organizations, you can access resources from the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov for further information. Participating in clinical trials may also offer opportunities for involvement in research and access to potential treatments, so inquire about eligibility for ongoing studies.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
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 Jan 7, 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. 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.