NON RARE IN EUROPE: Ulcerative colitis

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Overview

Ulcerative colitis (UC) is a chronic inflammatory bowel disease (IBD) characterized by continuous inflammation and ulceration of the mucosal lining of the large intestine (colon) and rectum. The inflammation typically begins in the rectum and extends proximally in a continuous pattern, distinguishing it from Crohn's disease, which can affect any part of the gastrointestinal tract in a patchy distribution. UC primarily affects the gastrointestinal system but can also have extraintestinal manifestations involving the joints, skin, eyes, and liver (including primary sclerosing cholangitis). The disease follows a relapsing-remitting course, with periods of active inflammation (flares) alternating with periods of remission. Key symptoms include bloody diarrhea, rectal bleeding, urgency to defecate, tenesmus (a feeling of incomplete evacuation), abdominal pain and cramping, and fatigue. During severe flares, patients may experience weight loss, fever, and anemia. The severity of symptoms generally correlates with the extent of colonic involvement. Long-standing ulcerative colitis increases the risk of colorectal cancer, necessitating regular surveillance colonoscopies. The treatment landscape for ulcerative colitis includes several therapeutic approaches aimed at inducing and maintaining remission. First-line therapies include 5-aminosalicylic acid (5-ASA) compounds such as mesalazine for mild-to-moderate disease. Corticosteroids are used for acute flares but are not suitable for long-term maintenance. Immunomodulators (azathioprine, 6-mercaptopurine), biologic therapies (anti-TNF agents such as infliximab and adalimumab, anti-integrin agents such as vedolizumab, and anti-IL-12/23 agents such as ustekinumab), and Janus kinase (JAK) inhibitors (tofacitinib, upadacitinib) are used for moderate-to-severe disease. Surgical intervention with proctocolectomy and ileal pouch-anal anastomosis (IPAA) remains a curative option for refractory cases or when complications such as dysplasia or cancer arise.

Also known as:

Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Sep 2011

Remicade: FDA approved

For reducing signs and symptoms and inducing and maintaining clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy

FDAcompleted
Dec 2006

Colazal: FDA approved

Treatment of mildly to moderately active ulcerative colitis in patients 5 years of age and older. Safety and Effectiveness of Colazal beyond 8 weeks in children (ages 5-17) and 12 weeks in adults have not been established.

FDAcompleted

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

Treatments

3 available

PHYTONADIONE PHYTONADIONE

PHYTONADIONE· Dr.Reddy's Laboratories Inc■ Boxed Warning

hypoprothrombinemia secondary to factors limiting absorption or synthesis of Vitamin K, e.g., ulcerative colitis

Remicade

infliximab· Janssen Biotech, Inc.■ Boxed WarningOrphan Drug
For reducing signs and symptoms and inducing and maintaining clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an ina

For reducing signs and symptoms and inducing and maintaining clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy

Colazal

Balsalazide disodium· Salix Pharmaceuticals, Inc.Orphan Drug
Treatment of mildly to moderately active ulcerative colitis in patients 5 years of age and older. Safety and Effectiveness of Colazal beyond 8 weeks in children (ages 5-17) and 12 weeks in adults have

Treatment of mildly to moderately active ulcerative colitis in patients 5 years of age and older. Safety and Effectiveness of Colazal beyond 8 weeks in children (ages 5-17) and 12 weeks in adults have not been established.

No actively recruiting trials found for NON RARE IN EUROPE: Ulcerative colitis at this time.

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No specialists are currently listed for NON RARE IN EUROPE: Ulcerative colitis.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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 NON RARE IN EUROPE: Ulcerative colitis.

Search all travel grants →NORD Financial Assistance ↗

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Caregiver Resources

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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.

Common questions about NON RARE IN EUROPE: Ulcerative colitis

What is NON RARE IN EUROPE: Ulcerative colitis?

Ulcerative colitis (UC) is a chronic inflammatory bowel disease (IBD) characterized by continuous inflammation and ulceration of the mucosal lining of the large intestine (colon) and rectum. The inflammation typically begins in the rectum and extends proximally in a continuous pattern, distinguishing it from Crohn's disease, which can affect any part of the gastrointestinal tract in a patchy distribution. UC primarily affects the gastrointestinal system but can also have extraintestinal manifestations involving the joints, skin, eyes, and liver (including primary sclerosing cholangitis). The d

How is NON RARE IN EUROPE: Ulcerative colitis inherited?

NON RARE IN EUROPE: Ulcerative colitis follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

What treatment and support options exist for NON RARE IN EUROPE: Ulcerative colitis?

1 patient support program are currently tracked on UniteRare for NON RARE IN EUROPE: Ulcerative colitis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.