Hemoglobinopathy

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ORPHA:68364
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1Active trials42Specialists8Treatment centers

Where are you in your journey?

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

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Jul 2020TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Johns Hopkins All Children's Hospital — PHASE2

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Hemoglobinopathy.

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

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Phase 21 trial
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
Phase 2
Actively Recruiting Prior treatment eligible
PI: Deepak Chellapandian, MD (Johns Hopkins All Children's Hospital) · Sites: St. Petersburg, Florida · Age: 0–21 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

Showing 25 of 42View all specialists →
MK
Mira A Kohorst
ROCHESTER, MN
Specialist
1 Hemoglobinopathy publication
VW
Viroj Wiwanitkit
Specialist
2 Hemoglobinopathy publications
JO
Jennifer L Oliveira
Specialist
2 Hemoglobinopathy publications
DM
David M Manthei
SALT LAKE CITY, UT
Specialist
1 Hemoglobinopathy publication
SL
Shih-Hon Li
ANN ARBOR, MI
Specialist
1 Hemoglobinopathy publication
DK
David F Keren
ANN ARBOR, MI
Specialist
1 Hemoglobinopathy publication
CG
Carmen Gherasim
Specialist
1 Hemoglobinopathy publication
SS
Siva Arumugam Saravanaperumal
Specialist
1 Hemoglobinopathy publication
TK
Tejaswi Koganti
Specialist
1 Hemoglobinopathy publication
GJ
Garrett Jenkinson
Specialist
1 Hemoglobinopathy publication
MH
Molly S Hein
BERLIN, MA
Specialist
1 Hemoglobinopathy publication
CT
Christineil H Thompson
ROCHESTER, MN
Specialist
1 Hemoglobinopathy publication
LH
Linda Hasadsri
ROCHESTER, MN
Specialist
1 Hemoglobinopathy publication
JH
James D Hoyer
ROCHESTER, MN
Specialist
2 Hemoglobinopathy publications
TM
Theo Heller, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 8 active trials
SM
Swee Lay Thein, M.D.
BETHESDA, MD
Specialist
PI on 11 active trials
LP
Leshana St. Jean, PhD
Specialist
PI on 2 active trials
JM
Jane Hankins, MD, MS
MEMPHIS, TN
Specialist
PI on 4 active trials
RM
Rick M Fairhurst, M.D.
ROCKVILLE, MD
Specialist
PI on 5 active trials
SP
Staci M Peron, Ph.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 8 active trials
PM
Petrign FG Töndury, MD
Specialist
PI on 1 active trial
AM
Aniket Saha, MD
GREENVILLE, SC
Specialist
PI on 1 active trial1 Hemoglobinopathy publication
DM
Daniel S Tsze, MD, MPH
NEW YORK, NY
Specialist
PI on 1 active trial
MM
Markus Schmugge Liner, MD
Specialist
PI on 1 active trial
MM
Michael R DeBaun, MD, MPH
NASHVILLE, TN
Specialist
PI on 4 active trials

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 Hemoglobinopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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

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Latest news about Hemoglobinopathy

Disease timeline:

New trial: TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Di

Phase PHASE2 trial recruiting. Haploidentical Hematopoietic Cell Transplantation

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 Hemoglobinopathy

Are there clinical trials for Hemoglobinopathy?

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

Which specialists treat Hemoglobinopathy?

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