News

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

Heart surgery can be performed safely in people with cold agglutinin disease (CAD) by adopting a series of procedure adjustments, according to a case study. The study, “Cardiopulmonary bypass strategy in a patient with cold agglutinin of high thermal amplitude,” was published in the journal…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

People with cold agglutinin disease (CAD) have genetic alterations previously reported in patients with marginal zone lymphoma (MZL), suggesting the disorders might be related, study reports. The study, “Cold agglutinin–associated B-cell lymphoproliferative disease shows highly recurrent gains of chromosome 3 and 12 or 18,” was…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Long-term maintenance treatment with sutimlimab is safe, prevents red blood cell destruction, and increases the amount of hemoglobin in people with cold agglutinin disease (CAD), avoiding the need for blood transfusions. Those findings are detailed in the study “Inhibition of complement C1s in patients with cold…

Emotional resilience is a term used to describe how people respond to changes and their ability to adapt to stressful situations. Being resilient doesn’t mean that people don’t experience or feel the stress but it refers to how quickly they can “bounce back” or adapt to crises. Resilience can…

Cold agglutinin disease can lead to serious complications during pregnancy, according to a case study of two women with autoimmune hemolytic anemia (AIHA). The study’s researchers emphasized the importance of a complete molecular diagnosis of AIHA to estimate maternal and fetal risks, and to establish…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…