Bainbridge-Ropers syndrome

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ORPHA:352577OMIM:615485Q87.0
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2Active trials19Specialists8Treatment centers

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

Bainbridge-Ropers syndrome (also called BRPS) is a rare genetic condition caused by changes (mutations) in the ASXL3 gene. This gene plays an important role in how the body controls gene activity during development, especially in the brain. When the ASXL3 gene does not work properly, it leads to problems with brain development, growth, and other body systems. Children with Bainbridge-Ropers syndrome typically have significant intellectual disability and delayed development. They may be slow to reach milestones like sitting, walking, and talking. Many children have feeding difficulties starting in infancy, low muscle tone (feeling floppy), and distinctive facial features. Some children may also experience seizures. Growth is often poor, and many affected individuals are smaller than expected for their age. There is currently no cure for Bainbridge-Ropers syndrome. Treatment focuses on managing symptoms and supporting development. This may include physical therapy, occupational therapy, speech therapy, and special education services. Feeding support and seizure management may also be needed. Early intervention programs can help children reach their full potential. Because this condition affects multiple body systems, a team of specialists usually works together to provide the best care.

Also known as:

Key symptoms:

Severe intellectual disabilityDelayed speech and language developmentDelayed motor milestones like sitting and walkingLow muscle tone (floppiness)Feeding difficulties in infancyPoor weight gain and growthSeizures or epilepsyDistinctive facial featuresSmall head sizeSleep disturbancesRepetitive or self-stimulating behaviorsLimited or absent speechConstipation or other digestive issuesHigh pain toleranceHand flapping or other stereotypic movements

Clinical phenotype terms (41)— hover any for plain English
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
May 2025Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation

n-Lorem Foundation — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING
Sep 2017ASXL-Related Disorders Natural History Study

University of California, Los Angeles

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Bainbridge-Ropers syndrome.

2 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

2 recruitingView all trials with filters →
Other1 trial
ASXL-Related Disorders Natural History Study
Actively Recruiting
PI: Wen-Hann Tan, BMBS (Boston Children's Hospital) · Sites: Los Angeles, California

Specialists

19 foundView all specialists →
ST
Shan Tian
GADSDEN, AL
Specialist
1 Bainbridge-Ropers syndrome publication
AL
Amaia Lasa-Aranzasti
Specialist
1 Bainbridge-Ropers syndrome publication
BM
Bárbara Masotto
Specialist
1 Bainbridge-Ropers syndrome publication
AA
Anna Abulí
Specialist
1 Bainbridge-Ropers syndrome publication
IV
Irene Valenzuela
MOUNT VERNON, TX
Specialist
1 Bainbridge-Ropers syndrome publication
SL
Shengjie Ling
Specialist
1 Bainbridge-Ropers syndrome publication
YZ
Yiming Zhang
MARION, IL
Specialist
1 Bainbridge-Ropers syndrome publication
RH
Rui Hu
CHARLOTTE, NC
Specialist
1 Bainbridge-Ropers syndrome publication
DZ
Dongdong Zhang
Specialist
1 Bainbridge-Ropers syndrome publication
WG
Weitong Guo
Specialist
1 Bainbridge-Ropers syndrome publication
LT
Laura Trujillano
Specialist
1 Bainbridge-Ropers syndrome publication
MC
Mar Costa-Roger
Specialist
1 Bainbridge-Ropers syndrome publication
IC
Ivon Cuscó
Specialist
1 Bainbridge-Ropers syndrome publication
PF
Paula Fernandez-Alvarez
Specialist
1 Bainbridge-Ropers syndrome publication
AC
Anna Cueto-González
Specialist
1 Bainbridge-Ropers syndrome publication
WB
Wen-Hann Tan, BMBS
Specialist
PI on 6 active trials
LP
Loren Pena, MD, PhD
Specialist
PI on 1 active trial
VP
Vandana Shashi, MD, PhD
DURHAM, NC
Specialist
PI on 1 active trial
BM
Bianca Russell, MD
LOS ANGELES, CA
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 Bainbridge-Ropers syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Bainbridge-Ropers syndrome

Disease timeline:

New recruiting trial: ASXL-Related Disorders Natural History Study

A new clinical trial is recruiting patients for Bainbridge-Ropers 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 ASXL3 mutation does my child have, and what does it mean for their development?,What therapies should we start right away, and how often should they occur?,Should we be screening for seizures, and what signs should I watch for?,What feeding strategies or nutritional support do you recommend?,Are there any clinical trials or research studies we could participate in?,What is the recommended schedule for follow-up visits with each specialist?,What resources are available for communication support and assistive technology?

Common questions about Bainbridge-Ropers syndrome

What is Bainbridge-Ropers syndrome?

Bainbridge-Ropers syndrome (also called BRPS) is a rare genetic condition caused by changes (mutations) in the ASXL3 gene. This gene plays an important role in how the body controls gene activity during development, especially in the brain. When the ASXL3 gene does not work properly, it leads to problems with brain development, growth, and other body systems. Children with Bainbridge-Ropers syndrome typically have significant intellectual disability and delayed development. They may be slow to reach milestones like sitting, walking, and talking. Many children have feeding difficulties startin

How is Bainbridge-Ropers syndrome inherited?

Bainbridge-Ropers syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Bainbridge-Ropers syndrome typically begin?

Typical onset of Bainbridge-Ropers syndrome is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Bainbridge-Ropers syndrome?

Yes — 2 recruiting clinical trials are currently listed for Bainbridge-Ropers syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Bainbridge-Ropers syndrome?

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