Tyrosinemia

Last reviewed

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Who is this for?
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3FDA treatments4Specialists8Treatment 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

Tyrosinemia is treated with 4 medications in our database, including ORFADIN, NITYR, Nitisinone, Orfadin. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Swedish Orphan Biovitrum AB (publ), Cycle, Swedish Orphan Biovitrum. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Tyrosinemia treatment below.

Inheritance

Autosomal recessive

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

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

NORD ↗

Treatments

3 available

NITYR

Nitisinone· Cycle

* Patient Copay Amount: $0 co-pay for eligible patients * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Benefits may be limited to commercially insured patients

ORFADIN

Nitisinone· Swedish Orphan Biovitrum AB (publ)

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi

Nitisinone

Nitisinone· Swedish Orphan Biovitrum

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi

No actively recruiting trials found for Tyrosinemia at this time.

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

Search ClinicalTrials.gov ↗Join the Tyrosinemia community →

Specialists

4 foundView all specialists →
GD
Gary Hubbard, Dr
Birmingham
Specialist

Rare Disease Specialist

PI on 1 active trial
AM
Anita MacDonald
Birmingham
Specialist

Rare Disease Specialist

PI on 10 active trials
CS
C. Ronald Scott
SEATTLE, WA
Specialist
PI on 1 active trial
TA
Tawhida Y Abdelghaffar
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 Tyrosinemia.

Search all travel grants →NORD Financial Assistance ↗

Community

Open TyrosinemiaForum →

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

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Latest news about Tyrosinemia

No recent news articles for Tyrosinemia.

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.

Common questions about Tyrosinemia

What is Tyrosinemia?

Tyrosinemia is treated with 4 medications in our database, including ORFADIN, NITYR, Nitisinone, Orfadin. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Swedish Orphan Biovitrum AB (publ), Cycle, Swedish Orphan Biovitrum. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Tyrosinemia treatment below.

How is Tyrosinemia inherited?

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

Which specialists treat Tyrosinemia?

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

What treatment and support options exist for Tyrosinemia?

3 FDA-approved treatments are currently tracked on UniteRare for Tyrosinemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.