Overview
Also known as:
FDA & Trial Timeline
6 eventsAzacitidine: FDA approved
Treatment of pediatric patients aged one month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML)
Indicated for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.
National Cancer Institute (NCI) — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableInqovi
Indicated for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British sub…
Indicated for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersChildren's Hospital Colorado
📍 Aurora, Colorado
👤 Zachary Grinspan, MD
Arkansas Children's Hospital
📍 Little Rock, Arkansas
Children's Hospital of Orange County
📍 Orange, California
👤 Richard Neibeger, MD
Children's National Medical Center
📍 Washington D.C., District of Columbia
👤 Richard Neibeger, MD
Connecticut Children's Medical Center
📍 Hartford, Connecticut
University of Miami Miller School of Medicine-Sylvester Cancer Center
📍 Miami, Florida
👤 Ann (Annie) W Silk
Harvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
Travel Grants
No travel grants are currently matched to Juvenile myelomonocytic leukemia.
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Start the conversation →Latest news about Juvenile myelomonocytic leukemia
Disease timeline:
New trial: Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Phase PHASE2 trial recruiting. Bone Marrow Aspiration and Biopsy
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 Juvenile myelomonocytic leukemia
Are there clinical trials for Juvenile myelomonocytic leukemia?
Yes — 1 recruiting clinical trial is currently listed for Juvenile myelomonocytic leukemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Juvenile myelomonocytic leukemia?
25 specialists and care centers treating Juvenile myelomonocytic leukemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.