W syndrome

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ORPHA:2804OMIM:311450Q87.8
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23Specialists8Treatment centers

Where are you in your journey?

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

W syndrome is an extremely rare genetic condition that affects multiple body systems. It was first described in the medical literature and is named after the initial letter of the surname of the family in which it was originally identified. The syndrome is characterized by a combination of features including intellectual disability, distinctive facial features, and skeletal abnormalities. Affected individuals may have a broad or flat nasal bridge, widely spaced eyes, and other facial differences. Skeletal problems can include short stature and abnormalities of the bones in the hands and feet. Some individuals may also have hearing loss and eye abnormalities. Because W syndrome is so rare, with only a handful of cases reported in the medical literature, knowledge about the full range of symptoms and the best approaches to treatment remains limited. There is currently no cure for W syndrome. Treatment focuses on managing individual symptoms and may involve a team of specialists. Early intervention services, including speech therapy, physical therapy, and special education support, can help affected children reach their full potential. Regular monitoring by healthcare providers is important to address any new symptoms that may arise over time.

Also known as:

Key symptoms:

Intellectual disabilityShort statureDistinctive facial featuresWidely spaced eyesBroad or flat nasal bridgeBone and skeletal abnormalitiesAbnormalities of the hands or feetHearing lossEye abnormalitiesDelayed developmentSpeech difficulties

Clinical phenotype terms (25)— hover any for plain English
Alternating esotropiaHP:0001137Metatarsus adductusHP:0001840Radial bowingHP:0002986Agenesis of maxillary central incisorHP:0006293Broad uvulaHP:0010809
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
Apr 20222177GCCC:ID'Ing tx Targets and Biomarkers for Ocular Surface Disease in Pt w/ oGVHD

University of Maryland, Baltimore

TrialRECRUITING
Sep 2021Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft

Stanford University — PHASE1

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for W syndrome.

View clinical trials →

No actively recruiting trials found for W syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the W syndrome community →

Specialists

23 foundView all specialists →
LA
Lowell Anthony
LEXINGTON, KY
Specialist
PI on 1 active trial4 W syndrome publications
DM
David Sallman, M.D.
TAMPA, FL
Specialist
PI on 1 active trial
DM
David Russell, MD
Specialist
PI on 3 active trials
PG
Peter L Greenberg
PALM DESERT, CA
Specialist
PI on 1 active trial2 W syndrome publications
PM
Paul Orchard, MD
MINNEAPOLIS, MN
Specialist
PI on 15 active trials
MM
Mary Horowitz, MD, MS
CHICAGO, IL
Specialist
PI on 10 active trials
RM
Robert Lowsky, MD
STANFORD, CA
Specialist
PI on 2 active trials
LM
Leland Metheny, MD
CLEVELAND, OH
Specialist
PI on 6 active trials1 W syndrome publication
WW
Wen-Kai Weng
STANFORD, CA
Specialist
PI on 4 active trials15 W syndrome publications
AM
Anna Pawlowska, MD
DUARTE, CA
Specialist
PI on 3 active trials
EM
Everett Meyer
STANFORD, CA
Specialist
PI on 2 active trials15 W syndrome publications
LM
Lori Muffly
STANFORD, CA
Specialist
PI on 1 active trial
HM
Harriette R Mogul, MD MPH
VALHALLA, NY
Specialist
PI on 1 active trial
EM
Everett Meyer, MD,PhD
STANFORD, CA
Specialist
PI on 1 active trial
DM
David LEPPERT, MD
Specialist
PI on 3 active trials
IS
Inderpaul si Sehgal
Specialist
PI on 1 active trial5 W syndrome publications
TM
Tian Yi Zhang, MD
Specialist
PI on 1 active trial
MP
Marcus Chen, MD, PhD
Specialist
PI on 1 active trial
SM
Sarah Sunshine, MD
CHICAGO, IL
Specialist
PI on 1 active trial
AD
Amin A Mohammad, Ph. D
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 W syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

Open W syndromeForum →

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

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Latest news about W syndrome

Disease timeline:

New recruiting trial: Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft

A new clinical trial is recruiting patients for W syndrome

New recruiting trial: 2177GCCC:ID'Ing tx Targets and Biomarkers for Ocular Surface Disease in Pt w/ oGVHD

A new clinical trial is recruiting patients for W syndrome

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.What specific features of my child's condition led to the diagnosis of W syndrome?,Is genetic testing available that could confirm the diagnosis or identify the specific gene involved?,What therapies and early intervention services do you recommend?,How often should my child have hearing and vision screenings?,Are there any skeletal issues we should monitor, and will my child need orthopedic care?,What is the expected developmental outlook for my child?,Can you refer us to a genetic counselor to discuss family planning and recurrence risk?

Common questions about W syndrome

What is W syndrome?

W syndrome is an extremely rare genetic condition that affects multiple body systems. It was first described in the medical literature and is named after the initial letter of the surname of the family in which it was originally identified. The syndrome is characterized by a combination of features including intellectual disability, distinctive facial features, and skeletal abnormalities. Affected individuals may have a broad or flat nasal bridge, widely spaced eyes, and other facial differences. Skeletal problems can include short stature and abnormalities of the bones in the hands and feet.

How is W syndrome inherited?

W syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does W syndrome typically begin?

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

Which specialists treat W syndrome?

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