Hemochromatosis

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1Active trials16Specialists8Treatment centers

Where are you in your journey?

UniteRare data is compiled from authoritative primary sources (FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, NORD), then processed through automated and AI-assisted extraction pipelines.
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What is Hemochromatosis?

Hemochromatosis is treated with 12 medications in our database, including Deferasirox, Deferasorox, Deferasirox oral, Exjade, TERUFLEX BLOOD with Diversion Blood Sampling Arm 70 mL CPDA-1, and 7 more. 9 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Novartis, Terumo, Chiesi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hemochromatosis 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
NORD ↗

FDA & Trial Timeline

1 event
Jan 2001Treatment of Hemochromatosis

National Institutes of Health Clinical Center (CC) — PHASE2

TrialACTIVE NOT RECRUITING

Data is compiled from FDA regulatory filings and ClinicalTrials.gov, then processed through automated extraction; event classifications and dates may occasionally be misclassified. Verify against the linked FDA filing or trial record before clinical decisions. Updated periodically.

Treatments

0 FDA-approved · 9 other tracked

Source: openFDA + DailyMed · NDA / BLA labels with structured indications · refreshed weekly

Other tracked therapies (9)

Medications tracked in our therapeutics database that are not FDA-approved for Hemochromatosis via a labelled indication. May include investigational, off-label, or supportive therapies. Always verify with a clinician before use.

DEFERASIROX

Deferasirox· NovartisNot FDA-labelled for Hemochromatosis

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Jadenu

deferasirox· NovartisNot FDA-labelled for Hemochromatosis

* Patient Copay Amount: $0 (Medications are provided free of cost to eligible patients) * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Must reside in the Unite

Deferiprone

deferiprone· ChiesiNot FDA-labelled for Hemochromatosis

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Deferoxamine

DEFEROXAMINE MESYLATE· NovartisNot FDA-labelled for Hemochromatosis

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

TERUFLEX BLOOD with Diversion Blood Sampling Arm 70 mL CPDA-1

· TerumoNot FDA-labelled for Hemochromatosis

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Deferasirox oral

Deferasirox· NovartisNot FDA-labelled for Hemochromatosis

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Deferasorox

Deferasirox· NovartisNot FDA-labelled for Hemochromatosis

* Patient Copay Amount: $0 (Medications are provided free of cost to eligible patients) * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Must reside in the Unite

Exjade

deferasirox· NovartisNot FDA-labelled for Hemochromatosis

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Deferasirox Oral Granules

· NovartisNot FDA-labelled for Hemochromatosis

* 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

Clinical Trials

1 recruitingView all trials with filters →

Source: ClinicalTrials.gov · synced daily · phases, status, and PI names normalized at ingest

Phase 21 trial
Treatment of Hemochromatosis
Phase 2
Active — not recruiting
PI: Leonard N Chen, M.D. (National Institutes of Health Clinical Center (CC)) · Sites: Bethesda, Maryland · Age: 18+ years

Specialists

16 foundView all specialists →

Source: NPI Registry + PubMed · trial PI roles cross-referenced with ClinicalTrials.gov · ranked by match score (publications + PI activity + community signal)

SK
Shawn F Kane
ANCHORAGE, AK
Specialist

Specialty unconfirmed

University of North Carolina

Last updated · Apr 20261 Hemochromatosis publication
CR
Caroline Roberts
Specialist

Specialty unconfirmed

University of North Carolina

Last updated · Apr 20261 Hemochromatosis publication
PA
Paul C Adams
Specialist

Specialty unconfirmed

Western University

Last updated · Apr 20263 Hemochromatosis publications
DG
Domenico Girelli
Specialist

Specialty unconfirmed

University of Verona and Azienda Ospedaliera Universitaria Integrata Verona

Last updated · Apr 20262 Hemochromatosis publications
FB
Fabiana Busti
Specialist

Specialty unconfirmed

University of Verona and Azienda Ospedaliera Universitaria Integrata Verona

Last updated · Apr 20262 Hemochromatosis publications
JR
John D Ryan
Specialist

Specialty unconfirmed

Beaumont Hospital

Last updated · Apr 20261 Hemochromatosis publication
RP
Ryan Paulus, DO
CHAPEL HILL, NC
Specialist

Specialty unconfirmed

University of North Carolina

Last updated · Apr 20261 Hemochromatosis publication
WP
William C Palmer
Specialist

Specialty unconfirmed

Mayo Clinic Florida

Last updated · Apr 20261 Hemochromatosis publication
FS
Fernando F Stancampiano, MD
JACKSONVILLE, FL
Specialist

Specialty unconfirmed

Mayo Clinic Florida

Last updated · Apr 20261 Hemochromatosis publication
JO
John K Olynyk
Specialist

Specialty unconfirmed

Fiona Stanley Fremantle Hospital Group

Last updated · Apr 20261 Hemochromatosis publication
GR
Grant A Ramm
Specialist

Specialty unconfirmed

Fiona Stanley Fremantle Hospital Group

Last updated · Apr 20261 Hemochromatosis publication
EC
Elizabeth Cabrera
Specialist

Specialty unconfirmed

Northwestern University Feinberg School of Medicine

Last updated · Apr 20261 Hemochromatosis publication
GC
Gonzalo Crespo
Specialist

Specialty unconfirmed

Hospital Clinic

Last updated · Apr 20261 Hemochromatosis publication
LV
Lisa B VanWagner
DALLAS, TX
Specialist

Specialty unconfirmed

University of Texas Southwestern Medical Center

Last updated · Apr 20261 Hemochromatosis publication
GM
Giacomo Marchi
Specialist

Specialty unconfirmed

University of Verona and EuroBloodNet Referral Center for Iron Disorders

Last updated · Apr 20261 Hemochromatosis publication
LM
Leonard N Chen, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

National Institutes of Health Clinical Center

Last updated · Mar 2026PI on 3 active trials

Treatment Centers

8 centers

Source: NORD Rare Disease Centers + NIH Undiagnosed Diseases Network (UDN) · centers verified active within last 12 months

⚗️ Trial Site

National Institutes of Health Clinical Center

📍 Bethesda, Maryland

👤 Payal P Khincha, M.D.

👤 Christopher Grunseich, M.D.

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Boston Children's Hospital Rare Disease Program

Boston Children's Hospital

📍 Boston, MA

👤 Boston Children's Hospital Rare Disease Program

🏨 Children's

Children's Hospital Colorado Rare Disease Program

Children's Hospital Colorado

📍 Aurora, CO

👤 Boston Children's Hospital Rare Disease Program

🏨 Children's

Ann & Robert H. Lurie Children's Hospital Genetics

Lurie Children's Hospital

📍 Chicago, IL

👤 Boston Children's Hospital Rare Disease Program

🏥 NORD

Cincinnati Children's Hospital Medical Center

Cincinnati Children's

📍 Cincinnati, OH

👤 Boston Children's Hospital Rare Disease Program

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

Travel Grants

No travel grants are currently matched to Hemochromatosis.

Search all travel grants →NORD Financial Assistance ↗

Community

Open HemochromatosisForum →

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

Start the conversation →

Latest news about Hemochromatosis

Source: PubMed + NIH RePORTER + openFDA + clinical-journal RSS · last 30 days · disease-tagged at ingest by AI extraction with human QC

Disease timeline:

New trial: Treatment of Hemochromatosis

Phase PHASE2 trial recruiting. Phlebotomy

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 Hemochromatosis

What is Hemochromatosis?

Hemochromatosis is treated with 12 medications in our database, including Deferasirox, Deferasorox, Deferasirox oral, Exjade, TERUFLEX BLOOD with Diversion Blood Sampling Arm 70 mL CPDA-1, and 7 more. 9 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Novartis, Terumo, Chiesi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hemochromatosis treatment below.

Are there clinical trials for Hemochromatosis?

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

Which specialists treat Hemochromatosis?

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

What treatment and support options exist for Hemochromatosis?

9 FDA-approved treatments and 2 patient support programs are currently tracked on UniteRare for Hemochromatosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.

Frequently asked questions about Hemochromatosis

Auto-generated from canonical disease facts (Orphanet, OMIM, ClinicalTrials.gov, openFDA, NPPES). Not a substitute for clinical guidance.

  1. What is Hemochromatosis?

    Hemochromatosis is a rare disease catalogued in international rare-disease ontologies. It is typically inherited as variable. Age of onset is generally variable. For verified primary sources, see the UniteRare Hemochromatosis page.

  2. How is Hemochromatosis inherited?

    Hemochromatosis follows variable inheritance. Genetic counseling is recommended for affected families to understand recurrence risk in offspring and the likelihood of unaffected siblings being carriers. Variants in the underlying gene(s) may be identified via clinical genetic testing.

  3. Are there FDA-approved treatments for Hemochromatosis?

    Yes — UniteRare tracks 9 FDA-approved treatments with indications relevant to Hemochromatosis. Each entry includes prescribing information, orphan-drug-designation status where applicable, and the FDA application number for verification.

  4. Are there clinical trials recruiting for Hemochromatosis?

    UniteRare currently lists 1 clinical trial relevant to Hemochromatosis sourced from ClinicalTrials.gov. Each trial entry includes recruitment status, eligibility criteria summary, principal-investigator information, and study locations. Patients should discuss eligibility with their healthcare provider before enrolling.

  5. How do I find a specialist for Hemochromatosis?

    UniteRare lists 16 verified clinicians with documented expertise in Hemochromatosis, sourced from ClinicalTrials.gov principal-investigator records, PubMed publication histories, and the NPPES NPI registry. Filter by state or browse our state-specific specialist pages for nearby options.

See full Hemochromatosis page for complete clinical details, sources, and verified-specialist listings.

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