Personalized Search
Displaying 1 - 20 of 878
Shane Pope talks about living with PNH, or Paroxysmal Nocturnal Hemglobinuria, in this recorded conversation with Leigh Clark. Listen or read the transcript below.
Podcast Episode last updated
.
Shaquilla Gordon explains her journey with the
bone marrow failure
bone marrow failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be……
Podcast Episode last updated
.
Saturday, October 24, 2026Conference Co-ChairsUma Borate, MBBSAlice Mims, MDAplastic Anemia/PNH Agenda 7:30 - 8:15 Breakfast and Registration8:15 - 8:45 Welcome 8:45 - 9:15
Bone Marrow Failure
Bone Marrow Failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are…
Conference Event last updated
.
AbstractIntroduction
Azacitidine
Azacitidine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Azacitidine is approved by the U…
-venetoclax (AZA-VEN) has become a standard…
Article last updated
.
ABSTRACTThe RACE study (NCT02009747) compared horse
antithymocyte globulin
antithymocyte globulin:
ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH.
(hATG…
Article last updated
.
AbstractPurpose: To evaluate the efficacy and safety of the cluster of differentiation 47-targeted antibody magrolimab plus
azacitidine
azacitidine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off,…
Article last updated
.
Learn from Dr. Richard Childs, RADM, as he explains the FDA approval of Omnisirge for treating patients with Severe
Aplastic Anemia
Aplastic Anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a…
Podcast Episode last updated
.
Patients with
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
,
paroxysmal nocturnal hemoglobinuria…
Conference Event last updated
.
CONFERENCE LOCATION:Heldrich Hotel 10 Livingston AvenueNew Brunswick, NJ 08901Parking: Complimentary hotel valet parking CONFERENCE AGENDA
Aplastic Anemia
Aplastic Anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek…
Conference Event last updated
.
VEXAS syndrome is a clonal hemato-inflammatory disorder impacting older, predominantly male patients, characterized by systemic inflammation and progressive
bone marrow failure
bone marrow failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and…
Article last updated
.
SummaryDonor cell-derived haematological neoplasms (DDHN) are rare disorders and currently do not have standardised diagnostic criteria and therapeutic management. International experts in allogeneic transplantation and haematological malignancies from Europe, the Americas, and Australia worked together on behalf of the EBMT Practice Harmonisation and Guidelines Committee to delineate a pragmatic…
Article last updated
.
HighlightsIn lower-risk MDS subgroup, trisomy 8 and 9 were more frequent in MDS-AID.MDS-AID had fewer tier 1 or 2 mutations than MDS-control or MDS-ALE.ASXL1 mutations were enriched in MDS-ALE within lower-risk MDS.No significant differences in OS or PFS versus MDS-control.IPSS-M was the only variable independently associated with outcomes. AbstractBackgroundAutoimmune features are…
Article last updated
.
The timing of TP53
mutation
mutation:
Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem cell in the bone marrow.
acquisition carries important prognostic implications for patients with myeloproliferative neoplasms (MPNs) and…
Article last updated
.
A blood cancer survivor debunks the movie myth of a quick cure, exposing the harsh, lasting side effects that remain after ringing the bell.One phrase I get tired of hearing lately is that something is a process. This word is often over-used and applies to anything from life crisis to learning how to cook! However, now I am going to used that phrase.Movies are fictional, and we all know…
Article last updated
.
TP53-mutated (TP53-mt)
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at…
(MDS) are…
Article last updated
.