Kisho

Kisho is an information platform, not a medical provider. Nothing on this site constitutes medical advice, diagnosis, or treatment recommendations. All content is aggregated from publicly available sources (including ClinicalTrials.gov, PubMed, FDA.gov, and Orphanet) and is provided for informational purposes only. Clinical trial eligibility, treatment decisions, and any health-related actions should always be discussed with a qualified healthcare professional. Kisho does not endorse any specific therapy, organization, or clinical trial. Terms of use · Privacy policy

congenital pseudoarthrosis of the tibia - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

congenital pseudoarthrosis of the tibia

MONDO:0017462

Also known as:congenital pseudarthrosis of the tibiacongenital pseudarthrosis of tibia

Find Clinical TrialsTrials

Overview

A condition characterized by non-union of the tibia, which is present at birth. It is usually associated with neurofibromatosis type 1.

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials1 registered · 1 recruiting

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for congenital pseudoarthrosis of the tibia. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for congenital pseudoarthrosis of the tibia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for congenital pseudoarthrosis of the tibia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

1 clinical trial registered, 1 recruiting. Interventions under study include procedural interventions and biologic therapy. Pipeline includes 1 PHASE3. Research is primarily industry-sponsored.

38 publications have been identified in PubMed for congenital pseudoarthrosis of the tibia. Research spans Clinical Trial Publication (29%), Case Report / Case Series (24%), and Review / Meta-Analysis (21%).

Research Type	Count	% of Total
Clinical study results	11

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 9:42 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:295018

GARD

Genetic and Rare Diseases Info Center

GARD:21203

Questions for Your Care Team

→Are any of the 1 active clinical trial a good fit for my situation?
→What is the current development status of autologous osteogenic cells cultured from adipose tissue derived stem cells embedded in extracellular matrix with demineralized bone matrix., and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

congenital pseudoarthrosis of the tibia

Overview

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications