Rare genetic tumor

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ORPHA:68336
Who is this for?
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1Active trials24Specialists8Treatment 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

Familial adenomatous polyposis (FAP) is a rare inherited condition that causes hundreds to thousands of small growths called polyps to form inside the large intestine (colon) and rectum. These polyps are not cancer at first, but if left untreated, they almost always turn into colorectal cancer — usually before age 40. FAP is also sometimes called adenomatous polyposis coli or classic FAP. The condition is caused by a change (mutation) in a gene called APC. Beyond the colon, FAP can also cause polyps in the stomach and small intestine, as well as non-cancerous growths in other parts of the body such as the bones, skin, and eyes. A milder form called attenuated FAP (AFAP) causes fewer polyps and a later cancer risk. Because the cancer risk is so high, most people with FAP need surgery to remove part or all of the colon. Regular monitoring and early treatment can save lives. With proper care, many people with FAP live full and meaningful lives, though lifelong medical follow-up is essential.

Key symptoms:

Hundreds or thousands of polyps (small growths) in the colon and rectumRectal bleeding or blood in the stoolDiarrhea or changes in bowel habitsStomach pain or crampingUnexplained weight lossFatigue and tirednessPolyps in the stomach or small intestineBenign (non-cancerous) bone growths called osteomasCysts under the skin (epidermoid cysts)Benign eye spots called CHRPE (congenital hypertrophy of the retinal pigment epithelium)Desmoid tumors (fibrous growths, often in the abdomen)Dental abnormalities such as extra teethIncreased risk of other cancers including thyroid, liver, and brain tumors

Inheritance

Autosomal dominant

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

Age of Onset

Juvenile

Begins in the teen years

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Feb 2026Safety and Efficacy of Tegavivint in Patients With Metastatic Colorectal Carcinoma

HonorHealth Research Institute — PHASE1, PHASE2

TrialRECRUITING
Nov 2021Marker Assisted Selective ThErapy in Rare Cancers: Knowledge Database Establishing registrY Asia

National Cancer Center, Japan

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Rare genetic tumor.

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

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →

Specialists

24 foundView all specialists →
AS
Ann (Annie) W Silk
Los Angeles, California
Specialist

Rare Disease Specialist

PI on 1 active trial
SA
Srivandana Akshintala
WASHINGTON, DC
Specialist
PI on 1 active trial
TB
Tommaso Pippucci, Biologist
Bologna
Specialist

Rare Disease Specialist

PI on 3 active trials
SM
Sharon A Savage, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 7 active trials
SP
Staci M Peron, Ph.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 8 active trials
MP
Marcelo Serra, PhD
Buenos Aires, Buenos Aires
Specialist

Rare Disease Specialist

PI on 1 active trial
AP
Anna Jansen, MD, PhD
Silver Spring, Maryland
Specialist

Rare Disease Specialist

PI on 2 active trials
MP
Maria Lourdes Posadas Martinez, PhD
Specialist
PI on 1 active trial
TM
Todd E. Druley, MD
SAINT LOUIS, MO
Specialist
PI on 1 active trial
RP
Rosalind A Eeles, PhD
Specialist
PI on 1 active trial
NP
Noboru Yamamoro, MD, PhD
Specialist
PI on 1 active trial
TD
Tobias Haack, Dr.
Specialist
PI on 2 active trials
YM
Yuta Maruki, MD
Specialist
PI on 1 active trial
TP
Takuji Okusaka, MD, PhD
Specialist
PI on 1 active trial
MM
Michael N Sack, M.D.
SPEARFISH, SD
Specialist
PI on 3 active trials
GM
Geoffrey Liu, MD, MSc
Specialist
PI on 1 active trial
KP
Kenichi Nakamura, MD, PhD
Specialist
PI on 1 active trial
OD
Olaf Rieß, Prof. Dr.
Specialist
PI on 2 active trials
RP
Rima Nabbout, MD, PhD
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 Rare genetic tumor.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Rare genetic tumor

Disease timeline:

New recruiting trial: Marker Assisted Selective ThErapy in Rare Cancers: Knowledge Database Establishing registrY Asia

A new clinical trial is recruiting patients for Rare genetic tumor

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.At what age should my child or I start having colonoscopies, and how often?,Which type of surgery is best for my situation, and when should it happen?,Should other members of my family be tested for the APC gene mutation?,What are the signs of desmoid tumors, and how would they be treated?,Are there medications that can help reduce my polyps, and are they right for me?,What other cancers should I be screened for, and how often?,Are there clinical trials or new treatments I should know about?

Common questions about Rare genetic tumor

What is Rare genetic tumor?

Familial adenomatous polyposis (FAP) is a rare inherited condition that causes hundreds to thousands of small growths called polyps to form inside the large intestine (colon) and rectum. These polyps are not cancer at first, but if left untreated, they almost always turn into colorectal cancer — usually before age 40. FAP is also sometimes called adenomatous polyposis coli or classic FAP. The condition is caused by a change (mutation) in a gene called APC. Beyond the colon, FAP can also cause polyps in the stomach and small intestine, as well as non-cancerous growths in other parts of the body

How is Rare genetic tumor inherited?

Rare genetic tumor follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Rare genetic tumor typically begin?

Typical onset of Rare genetic tumor is juvenile. Age of onset can vary across affected individuals.

Are there clinical trials for Rare genetic tumor?

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

Which specialists treat Rare genetic tumor?

24 specialists and care centers treating Rare genetic tumor are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.