Pseudohypoaldosteronism

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:444916
Who is this for?
Show terms as
1Active trials4Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Pseudohypoaldosteronism (PHA) is a rare condition where the body does not respond properly to a hormone called aldosterone. Aldosterone normally tells the kidneys to hold onto salt (sodium) and get rid of potassium. In PHA, the body ignores this signal, which causes dangerous salt loss and a buildup of potassium in the blood. There are two main types. Type 1 (PHA1) can be inherited and is present from birth. It comes in two forms: a milder kidney-limited form (sometimes called renal PHA1) and a more severe form that affects multiple organs including the lungs, sweat glands, and gut. Type 2 PHA (also called Gordon syndrome) is a different condition where potassium is too high but blood pressure is also elevated. Symptoms often appear in newborns and include poor feeding, dehydration, low sodium, and high potassium levels. Treatment focuses on replacing salt and managing potassium levels. With proper treatment, many patients — especially those with the milder form — can live relatively normal lives. The severe multi-organ form requires lifelong management and more intensive care.

Key symptoms:

Low sodium (salt) levels in the bloodHigh potassium levels in the bloodDehydrationPoor feeding or difficulty eating in newbornsFailure to gain weight or grow normallyLow blood pressureVomitingExtreme tiredness or lethargyRecurrent lung infections or breathing problems (in the severe multi-organ form)Salty-tasting skinSalt wasting through sweat, urine, and stool

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Jan 2024Transient Pseudohypoaldosteronism Affecting Children With Urinary Tract Malformation

University Hospital, Strasbourg, France

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Pseudohypoaldosteronism.

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
Transient Pseudohypoaldosteronism Affecting Children With Urinary Tract Malformation
Actively Recruiting
· Sites: Strasbourg · Age: 16 yrs

Specialists

4 foundView all specialists →
MM
Michael R Knowles, MD
CHAPEL HILL, NC
Specialist
PI on 1 active trial

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

Travel Grants

No travel grants are currently matched to Pseudohypoaldosteronism.

Search all travel grants →NORD Financial Assistance ↗

Community

Open PseudohypoaldosteronismForum →

No community posts yet. Be the first to share your experience with Pseudohypoaldosteronism.

Start the conversation →

Latest news about Pseudohypoaldosteronism

No recent news articles for Pseudohypoaldosteronism.

Follow this condition to be notified when news becomes available.

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 type of PHA does my child have, and what does that mean for their long-term outlook?,How much sodium supplement does my child need each day, and how do I adjust this during illness or hot weather?,What are the warning signs of a salt crisis, and when should I go to the emergency room?,Are there foods my child should avoid because of high potassium content?,Will my child need treatment for life, or is there a chance they will outgrow this?,Should other family members be tested for this condition?,Are there any clinical trials or new treatments we should know about?

Common questions about Pseudohypoaldosteronism

What is Pseudohypoaldosteronism?

Pseudohypoaldosteronism (PHA) is a rare condition where the body does not respond properly to a hormone called aldosterone. Aldosterone normally tells the kidneys to hold onto salt (sodium) and get rid of potassium. In PHA, the body ignores this signal, which causes dangerous salt loss and a buildup of potassium in the blood. There are two main types. Type 1 (PHA1) can be inherited and is present from birth. It comes in two forms: a milder kidney-limited form (sometimes called renal PHA1) and a more severe form that affects multiple organs including the lungs, sweat glands, and gut. Type 2 PHA

At what age does Pseudohypoaldosteronism typically begin?

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

Are there clinical trials for Pseudohypoaldosteronism?

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

Which specialists treat Pseudohypoaldosteronism?

4 specialists and care centers treating Pseudohypoaldosteronism are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.