Because patients need help in different ways, we offer a variety of support services. From the patient/family HelpLine and support networks to live conferences, online classes and volunteer events, our aim is to positively impact as many lives as we can. Our community members contribute to that goal by sharing their own inspirational Stories of Hope, supporting each other's need to stay positive and motivated.
Diseases like aplastic anemia, MDS, and PNH are rare and difficult to understand. Patients who learn about their condition can take an active role in their treatment by asking good questions of their medical team and advocating for their own needs
PNH occurs because of a genetic change in bone marrow
PNH, or Paroxysmal nocturnal hemoglobinuria is an ultra-rare bone marrow failure disease in which red blood cells break apart. Normal red blood cells have a shield of proteins that protect them from coming under attack by the body's own immune system. PNH occurs because that protein shield is missing. PNH is often associated with reduced bone marrow function (low blood counts) caused by aplastic anemia.
Aplastic anemia is a rare disease in which the bone marrow stops producing enough blood cells. Any blood cells the marrow does make are completely normal, but there are simply not enough of them to serve the body's needs. With prompt and proper care, most patients can be successfully treated.
MDS classification systems divide MDS into subtypes based on the results of blood and bone marrow tests. Scoring systems such as IPSS and IPSS-R have been developed to determine the severity of the MDS, probable survival term, and the risk of MDS developing into
A correct diagnosis is essential to getting the right treatment - a complicated problem for a rare disease for which there is no single test. Because doctors see so few MDS cases, they need to conduct a variety of tests to determine what the patient’s disease is and is not.
MDS, or Myelodysplastic syndromes, are a family of rare disorders in which the bone marrow fails to make enough healthy red blood cells, white blood cells or platelets. This is caused by bone marrow producing lots of underdeveloped, or immature, cells that have an abnormal shape, size or look. These are called blast cells. Most experts agree that MDS is a form of blood and bone marrow cancer.