Atresia of urethra

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1Active trials1Specialists8Treatment centers1Financial resources

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Overview

Atresia of the urethra is a rare birth defect where the urethra — the tube that carries urine from the bladder out of the body — is either completely closed off or severely narrowed. This means urine cannot drain normally before or after birth. The condition is also sometimes called urethral atresia or congenital urethral obstruction. It is present from birth and is one of the most severe forms of lower urinary tract obstruction. Because the urethra is blocked, urine builds up in the bladder and can back up into the kidneys while the baby is still developing in the womb. This causes the bladder to become very enlarged and can seriously damage the kidneys. It also reduces the amount of amniotic fluid (the fluid surrounding the baby), which is needed for the baby's lungs to develop properly. As a result, many affected babies are also born with underdeveloped lungs, a condition called pulmonary hypoplasia. Treatment requires urgent surgery after birth to create a way for urine to drain. In some cases, doctors may attempt to intervene before birth (fetal intervention) to relieve the blockage and protect kidney and lung development. The overall outlook depends heavily on how much kidney and lung damage occurred before birth. This is a life-threatening condition that requires care from a specialized medical team.

Also known as:

Key symptoms:

No urine output after birthSeverely enlarged or distended bladderSwollen or enlarged abdomen in the newbornKidney damage or failure due to backed-up urineUnderdeveloped lungs (pulmonary hypoplasia)Low amniotic fluid during pregnancy (oligohydramnios)Difficulty breathing at birth due to underdeveloped lungsAbnormal appearance of the face and limbs related to low amniotic fluid (Potter sequence)Urinary tract infections in survivorsChronic kidney disease in survivors

Clinical phenotype terms (14)— hover any for plain English
MegacystisHP:0000021HydroureterHP:0000072Bladder fistulaHP:0004321Patent urachusHP:0010479Dilatation of the bladderHP:0010955Pulmonic regurgitationHP:0010444
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Oct 2017

Ascor: FDA approved

for the treatment of scurvy in adult and pediatric patients age 5 months and older for whom oral administrtaion is not possible, insufficient or contraindicated

FDAcompleted
Dec 2014Standardized Prenatal Clinical Care for LUTO

Baylor College of Medicine

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Atresia of urethra.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Standardized Prenatal Clinical Care for LUTO
Actively Recruiting
PI: Michael Belfort, MD PHD (Baylor College of Medicine) · Sites: Houston, Texas · Age: 02 yrs

Specialists

1 foundView all specialists →
MP
Michael Belfort, MD PHD
HOUSTON, TX
Specialist
PI on 2 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources

Ascor

McGuff Pharmaceuticals Inc.

Ascor — Contact McGuff Pharmaceuticals Inc.

Unverified — confirm before calling
Patient Assistance
Manufacturer Program
Accepting applications

Travel Grants

No travel grants are currently matched to Atresia of urethra.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Atresia of urethra

Disease timeline:

New recruiting trial: Standardized Prenatal Clinical Care for LUTO

A new clinical trial is recruiting patients for Atresia of urethra

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.How much kidney function does my child have, and what does that mean for their long-term health?,What surgical options are available, and what is the expected timeline for procedures?,Will my child need dialysis or a kidney transplant in the future?,Are there any dietary restrictions we need to follow to protect kidney function?,What signs should prompt us to go to the emergency room?,Is there any genetic testing recommended for our family, and what is the chance of this happening in a future pregnancy?,What specialists should be part of my child's long-term care team?

Common questions about Atresia of urethra

What is Atresia of urethra?

Atresia of the urethra is a rare birth defect where the urethra — the tube that carries urine from the bladder out of the body — is either completely closed off or severely narrowed. This means urine cannot drain normally before or after birth. The condition is also sometimes called urethral atresia or congenital urethral obstruction. It is present from birth and is one of the most severe forms of lower urinary tract obstruction. Because the urethra is blocked, urine builds up in the bladder and can back up into the kidneys while the baby is still developing in the womb. This causes the bladd

How is Atresia of urethra inherited?

Atresia of urethra follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Atresia of urethra typically begin?

Typical onset of Atresia of urethra is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Atresia of urethra?

Yes — 1 recruiting clinical trial is currently listed for Atresia of urethra on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Atresia of urethra?

1 specialists and care centers treating Atresia of urethra are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Atresia of urethra?

1 patient support program are currently tracked on UniteRare for Atresia of urethra. See the treatments and support programs sections for copay assistance, eligibility, and contact details.