Porphyria

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ORPHA:738E80.1E80.2
Who is this for?
Show terms as
1FDA treatments23Specialists8Treatment centers3Financial resources

Where are you in your journey?

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

Porphyria is treated with 3 medications in our database, including GIVLAARI, SCENESSE, Panhematin. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Alnylam, Clinuvel, Recordati. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Porphyria treatment below.

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

1 available

GIVLAARI

givosiran sodium· Alnylam

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Must have commercial insurance. Patients with Medicare, Medicaid,

No actively recruiting trials found for Porphyria at this time.

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

Search ClinicalTrials.gov ↗Join the Porphyria community →

Specialists

23 foundView all specialists →
AM
Amy K Dickey, MD
Specialist
PI on 1 active trial1 Porphyria publication
RM
Robert J. Desnick, Ph.D., M.D.
NEW YORK, NY
Specialist
PI on 1 active trial
JM
James P. Kushner, M.D.
SALT LAKE CITY, UT
Specialist
PI on 1 active trial
KM
Krzysztof Jaworski, MD
Specialist
PI on 1 active trial
HM
Herbert L. Bonkovsky, M.D.
WINSTON SALEM, NC
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

3 grants

Recordati Rare Diseases Patient Liaison Support

Recordati Rare Diseases

Eligibility is limited to individuals diagnosed with cystinosis who are at least five years of age. The program is designed to provide financial assistance for participation in curricular or extracurricular activities.

Apply ↗

The Assistance Fund: Porphyria

Apply ↗

Porphyria Financial Assistance Program

Apply ↗

Community

Open PorphyriaForum →

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

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Latest news about Porphyria

No recent news articles for Porphyria.

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 Porphyria

What is Porphyria?

Porphyria is treated with 3 medications in our database, including GIVLAARI, SCENESSE, Panhematin. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Alnylam, Clinuvel, Recordati. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Porphyria treatment below.

Which specialists treat Porphyria?

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

What treatment and support options exist for Porphyria?

1 FDA-approved treatment and 1 patient support program are currently tracked on UniteRare for Porphyria. See the treatments and support programs sections for copay assistance, eligibility, and contact details.