Empowering the MDS Community
Patient & Visitor
Explore comprehensive resources tailored for patients and their loved ones. Understand your diagnosis, access treatment information, and find support in every step of your journey. We're here to provide clarity, comfort, and a sense of community.
Who is the MDS Foundation?
We are a global non-profit advocacy organization building critical awareness of Myelodysplastic Syndromes (MDS) - a rare, often undetected blood cancer. The MDS Foundation has supported patients and their families as well as healthcare providers in the fields of MDS, AML and other related cancers for over 30 years.
The MDS FoundationWhat is MDS?
Myelodysplastic syndromes (MDS) are a group of bone marrow failure disorders. Healthy bone marrow produces immature blood cells — stem cells - that normally develop into mature, fully functional red blood cells, white blood cells, and platelets. In MDS, these stem cells may not mature and may accumulate in the bone marrow or they may have a shortened life span, resulting in fewer than normal mature blood cells in the circulation.
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January 13, 2025
Rigel Announces R289 Granted Orphan Drug Designation by the FDA for MDS
Rigel Announces R289 Granted Orphan Drug Designation by the FDA for MDS
Rigel Pharmaceuticals (Nasdaq: RIGL) announced that the FDA has granted Orphan Drug designation to its investigational therapy R289 for the treatment of myelodysplastic syndromes (MDS). R289, a dual inhibitor of IRAK1 and IRAK4, is currently under investigation in a Phase 1b study assessing its safety, tolerability, and early effectiveness in patients with lower-risk MDS who have not responded to prior treatments. The Orphan Drug designation underscores the need for new therapies in rare diseases like MDS and provides Rigel with potential benefits such as tax credits, reduced FDA fees, and possible market exclusivity once approved. R289 had previously received Fast Track designation from the FDA for treating transfusion-dependent lower-risk MDS.
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Materials Spotlight
Testimonials
The MDS Foundation has been a great resource for our patients. We held a successful local symposium last year and hope to do that again in the future.
Afaf O
MDSF is fantastic so glad to have you there.
Amy D
We have enjoyed working with the MDS Foundation for many years.
Richard L
MDSF is a terrific organisation, and we appreciate the relationship.
Daniel P
I refer people regularly to the website and patient support services. The MDS Foundation is a great resource.
Mark J
I and my patients are huge fans of the MDS Foundation!
Yazan M
We are excited to keep working with the MDS Foundation as an MDS Center of Excellence to provide our patients the highest quality and state of the art care.
Aditi S
I believe that the Understanding Your MDS booklets and Building Blocks of Hope, MDS and AML Editions are invaluable resources, and I would love to be able to give them to my newly diagnosed patients.
Sophia B
My overall experience with the MDS Foundation is excellent.
Suresh D
I reviewed information on the MDS Foundation website in various languages, and it is very impressive.
Suresh D
It has been a tremendous blessing to have the MDS Foundation’s Northwestern team helping us through our MDS journey.
Stephen and Pat F
Center of excellence is like a 4 star Michelin restaurant rating.
Robert S
The MDS Foundation can assist you in getting into a center of excellence for MDS quickly. It is a top priority.
Amy C
I have found the forum to be a great help.
Marjory B
In my opinion everyone with MDS should put their trust in center of excellence.
Michael
