Overview
Neonatal diabetes mellitus (NDM) is a rare form of diabetes that appears in the first six months of life. Unlike the more common type 1 diabetes, which is caused by the immune system attacking insulin-producing cells, neonatal diabetes is almost always caused by a genetic change that affects how the pancreas makes or releases insulin. Insulin is a hormone the body needs to move sugar from the blood into cells for energy. Without enough insulin, blood sugar levels rise dangerously high. Babies with NDM may be born smaller than expected, feed poorly, lose weight, and become very ill quickly. High blood sugar can cause excessive urination, dehydration, and in severe cases, a life-threatening condition called diabetic ketoacidosis (DKA). Some children also have other health problems depending on which gene is affected, such as muscle weakness, developmental delays, or problems with the thyroid or pancreas. There are two main types: transient NDM, where diabetes goes away on its own within months but may return later in life, and permanent NDM, where insulin treatment is needed lifelong. Treatment depends on the genetic cause. Many children with mutations in the KCNJ11 or ABCC8 genes can switch from insulin injections to oral sulfonylurea pills, which is a major improvement in quality of life. Early genetic testing is essential to guide the best treatment.
Key symptoms:
Very high blood sugar levelsExcessive thirstFrequent urination or very wet diapersPoor feeding or refusing to eatSlow weight gain or weight lossDehydrationExtreme tiredness or low energyIrritability or unusual fussinessSmall size at birth (intrauterine growth restriction)VomitingRapid breathing (sign of diabetic ketoacidosis)Developmental delays (in some genetic forms)Muscle weakness (in some genetic forms)Seizures (in some genetic forms, such as DEND syndrome)
Variable
Can be inherited in different ways depending on the underlying gene
Neonatal
Begins at or shortly after birth (first 4 weeks)
FDA & Trial Timeline
10 eventsUniversity of Health Sciences Lahore — NA
Institute of Mother and Child, Warsaw, Poland — NA
South China Normal University — NA
Erasme University Hospital
University of California, San Francisco — NA
The University of Queensland — PHASE4
Women and Infants Hospital of Rhode Island
University of Massachusetts, Worcester — NA
Oxford University Clinical Research Unit Indonesia — NA
Medical University of Graz
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Neonatal diabetes mellitus.
2 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Rare Disease Specialist
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 Neonatal diabetes mellitus.
Community
No community posts yet. Be the first to share your experience with Neonatal diabetes mellitus.
Start the conversation →Latest news about Neonatal diabetes mellitus
Disease timeline:
New recruiting trial: PRIOR Study (Pre-eclampsia Risk In Oocyte Recipients)
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: Beta-cell Response to Incretin Hormones in Cystic Fibrosis
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: EXtremely Early-onset Type 1 Diabetes EXtremely Early-onset Type 1 Diabetes (A Musketeers' Memorandum Study)
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: New Markers of Glycation to Predict Gestational Diabetes Mellitus and Macrosomia.
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: Continuous Glucose Monitoring and OGTT Screen for Cystic Fibrosis Related Diabetes in Cystic Fibrosis
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: Identification of Dysglycemia With Continuous Glucose Monitoring to Assess Clinical Evolution in Cystic Fibrosis
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: Gestational Diabetes and Health Outcomes in Mothers and Babies
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: Intrapartum Glucose Control and Risk of Neonatal Hypoglycemia
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: Danish Diabetes Birth Registry 2
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
New recruiting trial: Pasteurised Donor Human Milk Supplementation for Term Babies
A new clinical trial is recruiting patients for Neonatal diabetes mellitus
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.
Questions for your doctor
Bring these to your next appointment
- Q1.Which gene is causing my child's diabetes, and how does that affect the best treatment choice?,Is my child a candidate to switch from insulin to oral sulfonylurea pills, and what would that process look like?,Will my child's diabetes go away on its own, or will they need treatment for life?,Are there other health problems we should watch for based on the specific genetic cause?,What should I do if my child's blood sugar is dangerously low or high at home?,Should other family members be tested for the same genetic change?,What support resources are available for our family, including psychological support and patient groups?
Common questions about Neonatal diabetes mellitus
What is Neonatal diabetes mellitus?
Neonatal diabetes mellitus (NDM) is a rare form of diabetes that appears in the first six months of life. Unlike the more common type 1 diabetes, which is caused by the immune system attacking insulin-producing cells, neonatal diabetes is almost always caused by a genetic change that affects how the pancreas makes or releases insulin. Insulin is a hormone the body needs to move sugar from the blood into cells for energy. Without enough insulin, blood sugar levels rise dangerously high. Babies with NDM may be born smaller than expected, feed poorly, lose weight, and become very ill quickly. Hi
At what age does Neonatal diabetes mellitus typically begin?
Typical onset of Neonatal diabetes mellitus is neonatal. Age of onset can vary across affected individuals.
Are there clinical trials for Neonatal diabetes mellitus?
Yes — 2 recruiting clinical trials are currently listed for Neonatal diabetes mellitus on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Neonatal diabetes mellitus?
25 specialists and care centers treating Neonatal diabetes mellitus are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.