/
Neurology
Congenital Myasthenic Syndrome
4
Pipeline Programs↗
4
Active Trials↗
2
Companies Involved↗
Congenital Myasthenic Syndrome is an early-stage landscape with 4 programs across 2 companies. The most active players are Argenx. 1 program is in Phase 3.
Intelligence
Pipeline Metrics
Competitive Density
Pipeline Velocity
Mech. Concentration
Completion Rate
Attrition Rate
Competition
Competitive Landscape
Bubble size = clinical trials
Approved
Phase 3
Phase 2
Phase 1
Preclinical
Key Players
Top Companies Companies ranked by the number of pipeline programs they run for this disease.
Pipeline
Programs by Phase
Phase 2
No programs
Phase 31
Approved
No programs
Analysis
Modality Breakdown
2programs
Small molecule150%
Cell therapy150%
Modality classified for 75% of 4 pipeline programs.
Analysis
Trial Status
5
clinical trials
Recruiting2 · 40%
Approved for marketing1 · 20%
Not yet recruiting1 · 20%
Active, not recruiting1 · 20%
Clinical Trials
Trial Activity
Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution
Recruiting·TThe Foundation for Orthopaedics and Regenerative Medicine·Phase 1·NCT07226726·20 enrolled
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)