Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality

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ORPHA:86841OMIM:153550D46.6
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1FDA treatments1Active trials14Specialists8Treatment centers

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

Also known as:

Clinical phenotype terms— hover any for plain English:

Abnormal erythrocyte morphologyHP:0001877ThrombocytosisHP:0001894Macrocytic anemiaHP:0001972Erythroid hypoplasiaHP:0012133Abnormal megakaryocyte morphologyHP:0012143Bone marrow hypercellularityHP:0031020Megakaryocyte nucleus hypolobulationHP:0031385Acute myeloid leukemiaHP:0004808Bone marrow hypocellularityHP:0005528AnisocytosisHP:0011273Abnormal neutrophil morphologyHP:0011992Abnormality of bone marrow stromal cellsHP:0012129Multiple lineage myelodysplasiaHP:0012148Chronic infectionHP:0031035
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Mar 2018A Trial Testing Early vs Late Onset of EPO Alfa Treatment in Lower Risk MDS

Groupe Francophone des Myelodysplasies — PHASE3

TrialACTIVE NOT RECRUITING

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

Treatments

1 available

Rytelo

IMETELSTAT SODIUM· Geron Corporation
RYTELO is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over

RYTELO is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA)

Clinical Trials

1 recruitingView all trials with filters →
Phase 31 trial
A Trial Testing Early vs Late Onset of EPO Alfa Treatment in Lower Risk MDS
Phase 3
Active
PI: Sophie Park, Prof (University Hospital, Grenoble) · Sites: Amiens; Angers +37 more · Age: 1899 yrs

Specialists

14 foundView all specialists →
UP
Ulrich Germing, Prof.
Specialist
PI on 1 active trial
GF
Ghulam J Mufti, MB, DM, FRCP, FRCPath
Specialist
PI on 2 active trials
RK
Rami Komrokji
TAMPA, FL
Specialist
PI on 2 active trials
SC
Sante Cundari
Specialist
PI on 1 active trial
LM
Lars Möllgård
Specialist
PI on 1 active trial
LA
Lionel ADES
Specialist
PI on 1 active trial
MT
Marie-Bérengère TROADEC
Specialist
PI on 1 active trial
CC
Consuelo del Cañizo
Specialist
PI on 1 active trial
EB
Emmanuel Besa
PHILADELPHIA, PA
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 Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality.

Search all travel grants →NORD Financial Assistance ↗

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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 Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality

Are there clinical trials for Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality?

Yes — 1 recruiting clinical trial is currently listed for Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality?

14 specialists and care centers treating Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality?

1 patient support program are currently tracked on UniteRare for Myelodysplastic syndrome associated with isolated del(5q) chromosome abnormality. See the treatments and support programs sections for copay assistance, eligibility, and contact details.