Overview
Diphtheria is an acute infectious disease caused by toxin-producing strains of the bacterium Corynebacterium diphtheriae. It is not a genetic disorder but rather an acquired bacterial infection that primarily affects the mucous membranes of the upper respiratory tract (pharyngeal and laryngeal diphtheria) and, less commonly, the skin (cutaneous diphtheria). The hallmark of respiratory diphtheria is the formation of a tough, grayish-white pseudomembrane in the throat and tonsils, which can obstruct the airway and cause difficulty breathing, a barking cough, hoarseness, and stridor. Systemic absorption of the diphtheria toxin can lead to serious complications affecting the heart (myocarditis, cardiac arrhythmias, heart failure), the nervous system (cranial and peripheral neuropathies, including palatal paralysis and limb weakness), and the kidneys (tubular necrosis). Other symptoms include sore throat, low-grade fever, cervical lymphadenopathy, and neck swelling sometimes described as a 'bull neck' appearance. Diphtheria was once a leading cause of childhood mortality but has become rare in developed countries due to widespread vaccination with diphtheria toxoid (included in DTaP/Tdap vaccines). However, it remains endemic in parts of Africa, South and Southeast Asia, and other regions with low vaccination coverage. Treatment involves prompt administration of diphtheria antitoxin (DAT) to neutralize circulating toxin, along with antibiotic therapy (typically erythromycin or penicillin) to eradicate the organism and prevent transmission. Supportive care, including airway management and cardiac monitoring, is essential. The case fatality rate remains approximately 5–10% even with treatment, and is higher in young children and the elderly. Prevention through routine immunization remains the most effective strategy against this disease.
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsChangchun BCHT Biotechnology Co. — PHASE1
Institute of Medical Biology, Chinese Academy of Medical Sciences — PHASE1, PHASE2
CanSino Biologics Inc. — PHASE1
Canadian Immunization Research Network — PHASE4
Changchun BCHT Biotechnology Co. — PHASE3
GC Biopharma Corp — PHASE1, PHASE2
Universiti Putra Malaysia — NA
LG Chem — PHASE2, PHASE3
Institute of Medical Biology, Chinese Academy of Medical Sciences — PHASE4
Bandim Health Project — PHASE4
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Diphtheria.
17 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Diphtheria.
Community
No community posts yet. Be the first to share your experience with Diphtheria.
Start the conversation →Latest news about Diphtheria
Disease timeline:
New recruiting trial: Prospective Biological Study to Evaluate the Persistence of COVID-19 Vaccine and Other Vaccines'-Induced Immune Responses in Follicular Lymphoma Patients Undergoing Frontline Induction Immuno-chemotherapy and Anti-CD20 Maintenance
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: A Phase II/III Study to Evaluate the Immunogenicity, Safety and Lot-to-lot Consistency of LBVD, a Fully Liquid Hexavalent Diphtheria-tetanus-whole Cell Pertussis-hepatitis B-poliovirus-Haemophilus Influenzae Type b Conjugate (DTwP-HepB-IPV-Hib) Vaccine, in Healthy Infants as Primary Series
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: Immunogenicity and Safety of Diphtheria, Tetanus, Pertussis (DTaP) Vaccine in 3-month-old Infants
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: Concurrent Versus Sequential Administration of Tdap and RSV Vaccines in Pregnancy
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: Assessing Interventions to Increase Tdap Acceptance for Non-birthing Partners in Pregnancy
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: Phase I Clinical Trial of Diphtheria-Tetanus-acellular Pertussis Component Combined Vaccine
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: Clinical Trial of the Adsorbed Acellular Pertussis (Tricomponent) DTaP-Haemophilus Influenzae Type b (Conjugate)-ACYW135 Group Meningococcal (Conjugate) Combined Vaccine
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: A Phase I/II Study to Evaluate the Safety and Immunogenicity of GC3111B in Healthy Adults
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: Adsorption of Cell-free Diphtheria and Tetanus (Three-component) Combined With Vaccine Phase III Clinical Trial
A new clinical trial is recruiting patients for Diphtheria
New recruiting trial: Safety and Immunogenicity of DTaP-IPV/Hib Pentavalent Vaccine in Chinese 2-month-old Infants
A new clinical trial is recruiting patients for Diphtheria
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Diphtheria
What is Diphtheria?
Diphtheria is an acute infectious disease caused by toxin-producing strains of the bacterium Corynebacterium diphtheriae. It is not a genetic disorder but rather an acquired bacterial infection that primarily affects the mucous membranes of the upper respiratory tract (pharyngeal and laryngeal diphtheria) and, less commonly, the skin (cutaneous diphtheria). The hallmark of respiratory diphtheria is the formation of a tough, grayish-white pseudomembrane in the throat and tonsils, which can obstruct the airway and cause difficulty breathing, a barking cough, hoarseness, and stridor. Systemic abs
Are there clinical trials for Diphtheria?
Yes — 17 recruiting clinical trials are currently listed for Diphtheria on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Diphtheria?
25 specialists and care centers treating Diphtheria are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.