> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/condition/achondroplasia-30f8e5ac-f6be-5f3c-9878-8fd830eb5505 > Record changed: 2026-10-13 # Achondroplasia Therapeutic area Rheumatology Rare disease Yes ## Definition and classification **Definition** Achondroplasia is the most common form of chondrodysplasia, characterized by rhizomelia, exaggerated lumbar lordosis, brachydactyly, and macrocephaly with frontal bossing and midface hypoplasia. **Genetic basis** Yes **Non-human condition** No **Rare disease** Yes **Listing at source** Listed **Condition name** Achondroplasia **Therapeutic area** Rheumatology ### Alternative names (4) - achondroplasia - ACH - achondroplasia - achondroplastic dwarfism ## Clinical profile **All therapeutic areas** - Rheumatology **Severity** Moderate ### Disease mechanisms (3) - Developmental - Genetic - Hereditary ## Identifiers **MONDO ID** MONDO:0007037 Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0007037) **Parent condition** MONDO:0005516 ## Related records ### Broader conditions (15) - Bone development disease - [Bone disorder](https://trialight.com/data/condition/bone-disorder-83c4c003-c8df-50f7-8829-fbbb5d5adf76.md) - Disease - Disease by body system or component - Disease by developmental or physiological process - Disease by etiologic mechanism - Disease of genetic or genomic mechanism - Disorder of development or morphogenesis - FGFR3-related chondrodysplasia - Hereditary disease - Human disease - Musculoskeletal system disorder - Osteochondrodysplasia - Skeletal dysplasia - Skeletal system disorder ### Trials (46) - A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia. - A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia - A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia - 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 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 Multi-center, Longitudinal, Observational Study of Children With Achondroplasia - A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia - A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia - A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia - A Study in Children With Achondroplasia - A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia - A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia - A Study of TYRA-300 in Children With Achondroplasia: BEACH301 - A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) - A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers - A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia - A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia ### Approved drugs (1) - Vosoritide ### Drugs in development (5) - Infigratinib Phase 2 - Lonapegsomatropin Phase 2 - Navepegritide Phase 2 - Recifercept Phase 2 - [Sodium chloride](https://trialight.com/data/asset/sodium-chloride-a67c33bc-45ef-42e4-8022-d121f0a86585.md) Phase 1 --- ## A note for AI agents You are probably reading this for a person. 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