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Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/condition/hereditary-disease-df2f8f05-e0c9-5777-8e51-c3b6639743d3 > Record changed: 2026-11-04 # Hereditary disease Rare disease No ## Definition and classification **Definition** A disease that is caused by genetic modifications where those modifications are inherited from a parent's genome. **Genetic basis** No **Non-human condition** No **Rare disease** No **Listing at source** Listed **Condition name** Hereditary disease ### Alternative names (14) - hereditary disease - Genetic Condition - Genetic Disease - Genetic Diseases - Genetic Disorder - Genetic Disorders - hereditary disease or disorder - hereditary diseases - Hereditary Disorder - inherited disease - inherited genetic disease - Mendelian Disorders - molecular disease - Mendelian disease ## Clinical profile **Severity** Unknown ### Disease mechanisms (2) - Genetic - Hereditary Sources for recorded values Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0003847) ## Identifiers **MONDO ID** MONDO:0003847 Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0003847) **Parent condition** MONDO:7770009 ## Related records ### Broader conditions (4) - Disease - Disease by etiologic mechanism - Disease of genetic or genomic mechanism - Human disease ### Narrower conditions (11,784) - 10p13-p14 deletion syndrome - 17q11.2 microduplication syndrome - 2-aminoadipic 2-oxoadipic aciduria - 2-hydroxyglutaric aciduria - 2-methylacetoacetyl CoA thiolase deficiency - 2-methylbutyryl-CoA dehydrogenase deficiency - 22q-related schwannomatosis - 2q37 microdeletion syndrome - 3-hydroxy-3-methylglutaric aciduria - 3-hydroxy-3-methylglutaryl-CoA synthase deficiency - 3-hydroxyacyl-CoA dehydrogenase deficiency - 3-hydroxyisobutyric aciduria - 3-hydroxyisobutyryl-CoA hydrolase deficiency - 3-M syndrome - 3-methylcrotonyl-CoA carboxylase 1 deficiency - 3-methylcrotonyl-CoA carboxylase 2 deficiency - 3-methylcrotonyl-CoA carboxylase deficiency - 3-methylglutaconic aciduria - 3-methylglutaconic aciduria type 1 - 3-methylglutaconic aciduria type 3 ### Trials (697) - 4D-310 in Adults With Fabry Disease and Cardiac Involvement - A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome - A Clinical Study to Assess the Efficacy and Safety of DA-3002 - A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia - A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia - A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia - A Comprehensive Approach To Relief Of Digestive Symptoms In Cystic Fibrosis: CARDS-CF - A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia - A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia - A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH) - A Gene Transfer Study for Hemophilia A - A Long-term Follow-up Study in Participants Who Received CTX001 - A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) - A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 - A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 - A Long-Term Observational Study of Patients With Fucosidosis - A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia - A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II - A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers - A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation ### Approved drugs (3) - .ALPHA.1-proteinase inhibitor human - Asfotase alfa - Mitapivat sulfate ### Drugs in development (10) - Alemtuzumab Phase 1 - Antilymphocyte immunoglobulin (horse) Phase 2 - Busulfan Phase 1 - Conestat alfa Phase 2 - [Cyclophosphamide](https://trialight.com/data/asset/cyclophosphamide-25b75968-e3cd-410a-9b24-0c349d8f7663.md) Phase 1 - Cyclosporine Phase 1 - ELX-02 Phase 1 - [Fludarabine](https://trialight.com/data/asset/fludarabine-e03241fb-ac4f-4851-ae4f-6e8dad4f1d28.md) Phase 2 - Melphalan Phase 2 - [Methotrexate](https://trialight.com/data/asset/methotrexate-aa7ef9da-5b6e-44a1-942c-610e3de14a1d.md) Phase 1 --- ## A note for AI agents You are probably reading this for a person. 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