A clonal hematopoietic disorder characterized by dysplasia and ineffective hematopoiesis in one or more of the hematopoietic cell lines. The dysplasia may be accompanied by an increase in myeloblasts,...
Comprehensive, easy-to-understand information about this condition
How we create this content →Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Documentation on myelodysplastic syndrome is limited due to its relatively low prevalence, affecting only 1-9 in 100,000 people. This rarity restricts the scope of systematic clinical studies and comprehensive phenotype documentation. Additionally, the genetic basis of MDS is still being explored, which can complicate the characterization of the condition. Ongoing research aims to enhance understanding and improve patient outcomes.
To navigate myelodysplastic syndrome, consider consulting a hematologist with expertise in blood disorders, particularly those specializing in myelodysplastic syndromes. Resources such as the Myelodysplastic Syndromes Foundation (https://mds-foundation.org) can provide valuable support and information. Additionally, participating in patient registries or natural history studies may be beneficial for both you and the broader MDS community. Genetic counseling could also be advantageous, especially given the known genetic factors associated with the condition.
Currently, there are 610 active clinical trials investigating various aspects of myelodysplastic syndrome. While there are no orphan drug designations, the extensive number of clinical trials indicates a robust research landscape. Patients can explore these trials for potential participation through the ClinicalTrials.gov search link: https://clinicaltrials.gov/search?cond=myelodysplastic%20syndrome.
Actionable guidance for navigating care for myelodysplastic syndrome
To navigate myelodysplastic syndrome, consider consulting a hematologist with expertise in blood disorders, particularly those specializing in myelodysplastic syndromes. Resources such as the Myelodysplastic Syndromes Foundation (https://mds-foundation.org) can provide valuable support and information. Additionally, participating in patient registries or natural history studies may be beneficial for both you and the broader MDS community. Genetic counseling could also be advantageous, especially given the known genetic factors associated with the condition.
Consider asking your healthcare providers these condition-specific questions
Connect with organizations supporting the myelodysplastic syndrome community
Helpful links for rare disease information and support
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 8, 2026
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
Organizations with orphan designations or approved therapies for this disease
AbbVie Inc.
Other
Agios Pharmaceuticals, Inc.
Other
Amgen Inc
Other
Apogenix GmbH
Other
Aprea Therapeutics AB
Other
Ascentage Pharma Group Inc.
Other
Astex Pharmaceuticals, Inc.
Other
BeOne Medicines USA, Inc.
Other
BioSight, Ltd.
Other
Boehringer Ingelheim Pharmaceuticals, Inc.
Other
Celgene Corporation
Other
Celgene Corporation, a Bristol-Myers Squibb Company
Other
Clinigen Limited
Other
Cornerstone Pharmaceuticals, Inc.
Other
Curis, Inc.
Other
Cyclacel Limited
Other
Eisai Inc.
Other
Faron Pharmaceuticals Ltd.
Other
Gamida Cell Inc.
Other
Gemin X, Inc.
Other
Genzyme Corporation
Other
Geron Corporation
Other
Gilead Sciences, Inc.
Other
Halia Therapeutics, Inc.
Other
Hessian Pharmaceuticals, Inc.
Other
IVAX International GmbH
Other
Johnson & Johnson Pharmaceutical Research & Dev.
Other
Karyopharm Therapeutics, Inc.
Other
Kyntra Bio
Other
Legacy Pharma Inc.
Other
Minovia Therapeutics Ltd.
Other
Mirati Therapeutics, Inc
Other
NBI Pharmaceuticals, Inc.
Other
Neolpharma S.A.DE C.V.
Other
Onconova Therapeutics, Inc.
Other
Orphan Europe SARL
Other
Otsuka Pharmaceutical Development
Other
Pfizer, Inc.
Other
Pharmacia & Upjohn Company LLC
Other
RNR BioMedical Inc.
Other
Rigel Pharmaceuticals, Inc.
Other
Schering Corporation
Other
Seattle Genetics, Inc.
Other
Servier Pharmaceuticals LLC
Other
Silence Therapeutics GmbH
Other
Sparta Pharmaceuticals, Inc.
Other
Sumitomo Pharma Oncology, Inc.
Other
Syros Pharmaceuticals
Other
TLR Therapeutics, Inc.
Other
Taiho Oncology, Inc.
Other
Takeda Development Center Americas, Inc.
Other
Tarix Pharmaceuticals, Ltd.
Other
Teva Branded Pharmaceutical Products R&D, Inc.
Other
The Vaccine Company
Other